JUNE 2021 # 76
Upfront In My View Business Sitting Down With Drug delivery: microneedles How digital tech can Plugging the cell and gene Maik Jornitz – inspiring the that melt away improve lab efficiency skills gap next generation
08 18 42 – 44 50 – 51
AI Blasts Off
How supercomputers and algorithms will lead to a smart new era of drug development 22 – 31
www.themedicinemaker.com How Do You Balance Today’s Demands With Tomorrow’s Discoveries? Modify. Intensify. Amplify.
Partner with Sartorius to know where, when and how to intensify key processes, increasing throughput up to four times, doubling productivity and achieving flexibility to meet the demands of today while preparing for the discoveries of tomorrow.
Learn More About Process Intensification www.sartorius.com/process-intensification What Do We Do About Distribution? Editorial COVID-19 vaccine rollout is a lottery, with some countries faring much better than others
ver 70 percent of people over the age of 18 have received a first dose of COVID-19 vaccine in the UK. And over 40 percent have received Otwo doses. But when it comes to my country’s progress, I can’t say I’m particularly proud. The UK has received a steady supply of vaccines ever since the first approvals. Meanwhile in some low- income countries, even frontline healthcare workers have not yet been vaccinated. And in other wealthy European countries, there are still substantial numbers of vulnerable people who are not vaccinated. I have four relatives (aged 63–75) living overseas in EU countries; two have received a first dose but were turned away from booked appointments to receive their second doses because of a lack of supply (one month later and still no sign). The remaining pair have yet to receive their first dose... In a post written for the London School of Economics and Political Science’s EUROPP blog, Gareth Davies reported that EU states agreed early on that it would be “unacceptable” for some states to vaccinate their populations while others had nothing (1). Vaccines have been distributed in the EU on a per capita basis. Meanwhile, the UK is rapidly vaccinating people in their early thirties. Details of contracts between pharma manufacturers and various governments have not been References revealed, but it could be that UK contracts have clauses for 1. G Davies, EUROPP, “Has the UK really preferential supply. British nationalists have applauded the outperformed the EU on Covid-19 UK’s shrewd negotiating skills. Though I am grateful that I vaccinations?” (2021). Available at can be vaccinated, I’m not able to justify the fact that I am https://bit.ly/3vEOayR. receiving it ahead of others who need it more. Clearly, there 2. Office of the United States Trade are inequalities in global vaccine distribution. Representative, “Statement from Some believe that a waiver on COVID-19 vaccine patents Ambassador Katherine Tai on the Covid-19 may help. In a shock announcement last month, the Biden Trips Waiver,” (2021). Available at administration declared that the US would support such a https://bit.ly/3vGRMjV. move, despite the US previously opposing patent waivers (2). How Do You Balance Today’s Demands With But pharma industry advocates, including IFPMA, PhRMA and EFPIA, are strongly against interfering with patents – and Tomorrow’s Discoveries? claim that it could damage the global battle against the virus by distracting from the manufacturing and scale up challenges. I can’t say if patent waivers are the correct way forward. There Modify. Intensify. Amplify. are powerful arguments on both sides of the debate (see page 10). But I do know that desperate times call for desperate measures – and a global death toll of over 3.5 million sounds desperate to Partner with Sartorius to know where, when and how to intensify me. We need to thoroughly explore all avenues to see if they have key processes, increasing throughput up to four times, doubling merit – even if they may be controversial in some camps. productivity and achieving flexibility to meet the demands of today while preparing for the discoveries of tomorrow. Stephanie Sutton Editor Learn More About Process Intensification www.sartorius.com/process-intensification www.themedicinemaker.com Contents
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In My View
42 14 Lori Muffly believes that lessons learned during the pandemic will guide the future of stem cell transplatns
03 Editorial Upfront 16 We can find ways to improve What Do We Do on COVID-19 vaccines, says About Distribution? 06 The latest news, views Nigel Theobald By Stephanie Sutton and research – including frozen microneedles for delivering cell 18 According to Tola therapies, an award for Olorunnisola, there are myriad On The Cover sustainable machinery, and opportunities to improve lab thoughts on the concept of efficiency with digital tech AI takes off in the waiving patents for pharma industry COVID-19 vaccines 19 Mark Smith asks how we mitigate the risks and costs of choosing the wrong bioprocessing technologies ISSUE 76 - JUNE 2021
Feel free to contact any one of us: 25 [email protected] Content Team Editor - Stephanie Sutton James Strachan (Deputy Editor) Maryam Mahdi (Associate Editor) Commercial Team Publisher - Helen Conyngham Stacy Gaines (Business Development Manager, North America) Elen John-Morris (Business Development Executive) Design Team Head of Design - Marc Bird Hannah Ennis (Senior Designer) Charlotte Brittain (Designer)
Digital Team Digital Team Lead - David Roberts Peter Bartley (Digital Producer Web/Email) Abygail Bradley (Digital Producer Web/App) Audience Team Audience Growth Strategy Manager – Brice Agamemnon CRM & Compliance CRM & Compliance Manager - Tracey Nicholls Hayley Atiz (CRM Assistant) Commercial Support Team Internal Systems Manager - Jody Fryett Dan Marr (Campaign Reporting Analyst) Commercial Services Commercial Service and Social Media Manager - Matt Everett Alice Daniels-Wright (Video Project Manager) Jess Lines (Video and Project Support Coordinator) Lindsey Vickers (Sales Support Project Manager) Marketing Team Marketing Manager - Katy Pearson Feature Business Jo Baylay (Marketing Executive) Accounts Team Kerri Benson (Accounts Assistant) 22 Powered by AI 42 How to Plug the Skills Gap Emily Scragg (Accounts Apprentice) AI is changing the world of Investigating how lack of skills in Human Resources pharma drug development certain areas of the UK’s biotech Human Resource Manager - Tara Higby sector threaten potential growth Management Team Chief Executive Officer - Andy Davies Chief Operating Officer - Tracey Peers 46 Powering on Through Senior Vice President (North America) - Fedra Pavlou NextGen the Pandemic Financial Director - Phil Dale Commercial Director - Richard Hodson How is pharma dealing with the Content Director - Rich Whitworth 34 When the Chips are Down pandemic? We ask members of Find out how organ-on-chip our 2021 Power List. Change of address [email protected] technology is giving early Hayley Atiz, The Medicine Maker, Texere Publishing Limited, information on drug candidates Booths Park 1, Chelford Road, Knutsford, Cheshire, WA16 8GS, UK General enquiries www.texerepublishing.com | [email protected] +44 (0) 1565 745 200 | [email protected] 38 Fifty Functional Cures Report Distribution: The Medicine Maker (ISSN 2055-8201), The latest updates in advanced is published monthly by Texere Publishing Limited, Booths Park 1, Chelford Road, Knutsford, Cheshire, WA16 8GS, medicine, including CARs for 32 Global Support for Faster UK. Single copy sales £15 (plus postage, cost available on request [email protected]). Non-qualified annual solid tumors and a plethora of Formulation Development subscription cost is £110 plus postage new collaborations Reprints & Permissions – [email protected] The copyright in the materials contained in this publication and the typographical arrangement of this publication belongs to Texere Publishing Limited. No person may copy, modify, transmit, distribute, display, reproduce, publish, licence or create works from any part of this material or typographical arrangement, or otherwise use it, for any public or commercial Sitting Down With use without the prior written consent of Texere Publishing Limited. The names, publication titles, logos, images and presentation style appearing in this publication which identify Texere Publishing Limited and/or its products and services, including but without limitation Texere and The 50 Maik Jornitz, President and Medicine Maker are proprietary marks of Texere Publishing Limited. Nothing contained in this publication shall be deemed to confer on any person CEO of G-CON any licence or right on the part of Texere Publishing Limited with respect to any such name, title, logo, image or style. 6 Upfront Upfront
Research Results Trends Innovation That Last
Could zinc finger therapy hold the key to treating Alzheimer’s disease?
Imagine treating Alzheimer’s disease (AD) at the genetic level. Now consider researchers at Sangamo Therapeutics who have found that gene regulation therapy helps prevent the build-up of tau protein in the brain (1). “The tau protein is predominantly found in neurons. One of tau’s main functions is to stabilize the microtubules – small structures within the nerves – that are important to ensure impulses are conducted between neurons,” says Amy Pooler, Head of Neuroscience at Sangamo. “In AD, the tau protein is abnormal and tangles into various forms of toxic aggregates that disrupt communication between neurons, and potential to shift the treatment paradigm “Genome regulation is just one tool in ultimately lead to cell death.” for AD from symptom management to our genomic medicines kit. Though our The team decided to attempt lowering lasting cures,” says Pooler. collaborations in this space just kicked expression of the tau gene at the Sangamo CEO Jason Fontenot hopes off this past year, we are encouraged transcriptional level using gene-silencing the research can be applied to other by our progress in moving the zinc finger protein transcription factors debilitating neurological disorders – and research forward.” (ZFP-TFs) – and observed specific, the company has formed partnerships durable, and controlled reduction of with the likes of Biogen and Novartis to Reference endogenous tau production. look at how the approach can be applied 1. S Wegmann et al., Science Advances (2021). “With our ZFP-TFs, we have the to other therapeutic areas. Fontenot says, DOI: 10.1126/sciadv.abe1611
INFOGRAPHIC 60.1% White In 48 clinical trials for drugs 12.2% Black The Minority approved in the US in 2019: 18.5% Hispanic 5.6% Asian Report 9% of participants were black 0.7% American Indian 72% of participants were white Alaska Native 0.2%Native Hawaiian Are US clinical trials truly 18% of participants were Other Pacific Islander representative? Hispanic 2.8% Multiple Races Source: Parexel (2021). Available at 9% were Asian https://bit.ly/3uqw3eK. US demographics Upfront 7
Alexion – a company focused BUSINESS IN BRIEF on developing therapies for rare diseases. The US$39 billion deal Manufacturing violations, regulatory must be cleared by the authority A Tailored probes, and advertising campaigns... to allow AstraZeneca to expand what’s new in business? its offering for areas of unmet Solution need. The final decision will be • Due to alleged manufacturing issued by July 5. Reinventing drug delivery and records violations at Eli • The Pharmaceutical Research and with polymeric threads Lilly’s Branchburg, New Jersey Manufacturers of America has site, the US Department of launched a lobbying group against Researchers at the University of Justice (DOJ) has issued the President Biden’s plans for a patent California, Riverside have used a company a subpoena. During waiver for COVID-19 vaccines. technique known as electrospinning a 2019 inspection, it was found The organization has developed to develop a membrane made of that documents related to a digital advertising campaign polymeric threads that enables localized manufacturing processes were to protest the administration’s administration of drugs to sites in the missing. Reports also suggest proposed idea with Google body (1). The membrane is loaded with that some documents were advertisements that read, “Biden’s therapeutic material and embedded into altered to minimize the extent Harmful Vaccine Stance” and a hydrogel, and the researchers expose it of quality control issues. The “Biden’s Damaging IP Stance.” to shock waves that generate an electrical DOJ is now requesting that the • AbbVie faces questioning from charge, allowing for controlled release company, which is complying the US Senate for its tax record. of medicines. with the investigation, provide the The company, which is under According to the team, changing the documentation. investigation for its approach size of the threads achieves different • AstraZeneca is reportedly to drug pricing, is now under release kinetics. Smaller threads can holding discussions with the scrutiny as its American arm deliver drugs to deep tissues; larger US government to move its continues to lose money and threads administer medicines to COVID-19 vaccine production questions arise as to why it doesn’t subcutaneous tissues. The Riverside from Emergent BioSolutions’ plant pay more in tax. Senator Ron researchers expect that their method to another CDMO. Previously, Wyden penned a letter to the could help overcome various traditional a batch of J&J’s vaccine was company’s CEO inquiring about drug delivery challenges, including the contaminated with ingredients its current financial situation. nonspecific distribution of medicines from AstraZeneca’s. “It appears that AbbVie shifts throughout the body. • AstraZeneca is also being probed profits offshore while reporting a by the UK’s antitrust watchdog, domestic loss in the United States Reference the Competition and Markets to avoid paying US corporate 1. T Jariwala et al. ACS Appl. Bio Mater. Authority, for its acquisition of income taxes,” he said in the letter. (2021). DOI: 10.1021/acsabm.1c00232
Willingness Willingness to Have to Join a COVID-19 Vaccine COVID-19 Trial
Non white respondents White respondents 8 Upfront
Melt Away Delivery
Could frozen microneedles change the way we deliver cell therapies?
Researchers have designed and developed a microneedle technology to allow intradermal delivery of cell therapies for the treatment of skin disorders (1). The team, led by Xu Chenjie, Associate Professor at the Department of Biomedical Engineering at City University of Hong Kong, has produced cryogenic The needles measure less than 1 mm and of a cell therapy. “Another important microneedle patches (cryomicroneedles) deliver loaded living cells into the skin. design feature is the fact that cells packed that melt after delivering the therapeutic In healthy mice, cells delivered using into cryomicroneedles can be stored for payload. Traditional cell delivery methods the cryomicroneedle patch retained months, allowing easy transportation usually involve hypodermic needles and viability and proliferative capability. And and deployment,” says Xu. surgical intervention. in mice with subcutaneous melanoma The cryomicroneedles aren’t limited Commenting on the development, tumours, the delivery of ovalbumin- to cell therapy delivery; they can also Xu says, “The cryomicroneedles are pulsed dendritic cells using the be used to package, store, and deliver a manufactured by micromolding an cryomicroneedle approach led to higher variety of bioactive therapeutic agents, optimized cryogenic medium and antigen-specific immune responses and such as proteins, peptides, and vaccines pre-suspended therapeutic living cells slower tumor growth compared with – potential applications Xu hopes to at extremely low temperatures (-20 to intravenous and subcutaneous injections explore in future studies. -196℃). The original process relied on of the cells. the crystallization of ice, which held the Xu believes this approach would help Reference potential to kill the cells used, but the avoid complex processes such as cell 1. H Chang et al, Nature Biomedical cryogenic medium we developed helped harvesting and the preparation of cell- Engineering (2021). DOI: 10.1038/ avoid this issue by protecting the cells.” infusing solution during administration s41551-021-00720-1.
are subject to genetically “The European Time for a modified organism Commission recognized that (GMO) laws, which were time was of the essence when Change originally introduced to lifting GMO requirements regulate the production for COVID-19 vaccines How legislation can of food and protect the and treatments,” said Paige potentially hinder environment. According to the Bischoff, Alliance for Regenerative pharmaceutical progress coalition, the laws are also slowing Medicine’s Senior Vice President of the progress of advanced therapies Global Public Affairs, in a statement. A position paper published by an industry in clinical development. The opinion coalition cast scrutiny on current EU has been supported by the European Reference laws on advanced therapies (1). Under the Commission, who agrees that current 1. M Werner et al., Hum Gene Ther (2021). region’s legislation, advanced therapies guidance is hindering the field. DOI: 10.1089/hum.2021.058 Upfront 9
IMAGE OF THE MONTH A Celebration of Design
ACG secures an industry award for its sustainable machinery
ACG has scooped up an iF Design Award for its latest packaging machine, Karton X. The prize, which celebrates innovative product design, was awarded for the instrument’s human-machine interface, UX design, and smart connectivity. Though Karlton X was designed to pack blisters, bottles, tubes, vials, and ampoules in secondary packaging, a key focus for ACG was its sustainability. The company Research Accelerated produced the system using recyclable materials such as steel, aluminum, An international research coalition is using a new protocol to describe 3D structures and Corian. Karan Singh, Managing of SARS-CoV-2 molecules using nuclear magnetic resonance spectroscopy (NMR). Director of ACG, says, “For us, this Their NMR approach allows for active compounds that bind to viral proteins to be award is a validation that ACG is quickly identified, helping to fuel the next stages of pharmaceutical R&D. addressing the needs of the wide range Credit: Uwe Dettmar for Goethe-University Frankfurt, Germany of pharma manufacturing companies we work with. It acknowledges our Would you like your photo featured in Image of the Month? drive for innovation and reflects Send it to [email protected] our philosophy of designing and manufacturing high-quality, reliable, and innovative machines.”
QUOTE of the month
“Over the last few years, we have seen almost limitless opportunities for AI to disrupt all points of the healthcare spectrum – from medical records to drug discovery. But we need to focus on where we can have the most tangible impact now and what to work towards in the long term,” Brandon Allgood, Chief AI Officer, Valo Health, and Chairperson of The Alliance for Artificial Intelligence in Healthcare. https://bit.ly/3bVlOIW
www.themedicinemaker.com 10 Upfront
The Patent Waiver War
Is waiving patents for COVID-19 vaccines the right step forward?
The Biden administration has agreed to support negotiations at the World Trade Organization on waiving patent protections for COVID-19 vaccines – joining over 100 countries who already back the proposal. The hope is that this will address inequities and shortages in vaccine supply. However, there is a clear split in opinions on the matter across the world. Here, we round up governments opportunities to take action poured billions of US dollars of public money a selection of quotes from key stakeholders. for better collaboration in development, to support COVID-19 R&D, especially for production, and supply of COVID-19 drugs and vaccines. However, by and large, Those for medical tools without being restricted no conditions for access or affordability have by private industry’s interests and actions been included as a precondition to any of that Biden administration (1): “This is a and, crucially, would give governments all funding. Governments must attach strings global health crisis and the extraordinary available tools to ensure global access.” to any public money given for COVID-19 circumstances of the COVID-19 medical tools to guarantee that, if they pandemic call for extraordinary measures. UNAIDS (3): “We are in a race to vaccinate prove safe and effective, they are available The Administration believes strongly the majority of the world’s population to to everyone. Today some members have in intellectual property protections but, curb death tolls and before more potent admitted that some conditions had been in service of ending this pandemic, variants of COVID-19 emerge, rendering set on companies, but none of it goes far supports the waiver of those protections current vaccines ineffective. The faster we enough to ensure that IP rights assigned to for COVID-19 vaccines. We will actively can scale up global vaccine supply, the companies benefiting from taxpayer money participate in text-based negotiations at the faster we can contain the virus and the less do not abuse such rights down the line.” World Trade Organization (WTO) needed chance we will face a day when variants to make that happen.” prove resistant to existing vaccines. As the Those against United Nations Secretary-General Antonio MSF (2): “The temporary waiver would Guterres has said, “No one is safe until IFPMA (5): “A waiver is the simple but the apply to certain IP on COVID-19 medical everyone is safe.” The TRIPS waiver would wrong answer to what is a complex problem. tools and technologies until herd immunity enable the sharing of technologies, data, Waiving patents of COVID-19 vaccines is reached. It was originally proposed know-how, patents, and other intellectual will not increase production nor provide by India and South Africa in October property rights across the world.” practical solutions needed to battle this 2020 and is now officially backed by 58 global health crisis. On the contrary, it is sponsoring governments, with around 100 WTO (4): “The R&D of drugs is often likely to lead to disruption while distracting countries supporting the proposal overall. a joint multi-stakeholder effort benefiting from addressing the real challenges in Even after one year of this pandemic and from significant amounts of public taxpayer scaling up production and distribution 2.5 million deaths, we still see certain money. For COVID-19, the search for an of COVID-19 vaccines globally: namely, governments denying that removing effective treatment or vaccine is a global elimination of trade barriers, addressing monopolies on COVID-19 medical tools effort involving multiple actors – it is not bottlenecks in supply chains and scarcity of will help increase people’s access to needed the result of the pharmaceutical industry’s raw materials and ingredients in the supply treatments, vaccines, and tests going efforts alone. Governments and public chain, and a willingness by rich countries forward. The waiver proposal offers all funding agencies around the world have to start sharing doses with poor countries.” pda.org/EU/2021BioMan
2021 PDA BIOMANU- EFPIA (6): “This short-sighted and decision does nothing to address the real ineffectual decision by the Biden challenges to getting more shots in arms, administration puts the hard-won progress including last-mile distribution and limited FACTURING in fighting this terrible disease in jeopardy. availability of raw materials. These are the While we wholeheartedly agree with the real challenges we face that this empty CONFERENCE goal of protecting citizens around the world promise ignores. through vaccines, waiving patents will make “In the past few days alone, we’ve seen winning the fight against the coronavirus more American vaccine exports, increased even harder… Increasing capacity to production targets from manufacturers, new deliver doses to citizens around the world commitments to COVAX and unprecedented requires the skills and technical know-how aid for India during its devastating COVID-19 of the vaccine developer to bring on board surge. Biopharmaceutical manufacturers partner manufacturing organizations. You are fully committed to providing global simply cannot achieve this kind of capacity access to COVID-19 vaccines, and they are expansion by waiving patents and hoping collaborating at a scale that was previously that hitherto unknown factories around the unimaginable, including more than 200 world will turn their hand to the complex manufacturing and other partnerships to process of vaccine manufacture. A waiver date. The biopharmaceutical industry shares risks diverting raw materials and supplies the goal to get as many people vaccinated as away from well-established, effective supply quickly as possible, and we hope we can all chains to less efficient manufacturing sites re-focus on that shared objective.” where productivity and quality may be an issue. It opens the door to counterfeit vaccines References entering the supply chain around the world. 1. Office of the United States Trade Representative, Capacity expansion is only achievable “Statement from Ambassador Katherine Tai on the through voluntary, collaborative partnerships Covid-19 Trips Waiver,” (2021). Available at https:// between the innovators behind each vaccine bit.ly/3vGRMjV. and expert manufacturing partners. All 2. Medecins sans Frontieres, “Countries obstructing our focus should be on removing barriers COVID-19 patent waiver must allow negotiations to to collaboration, ensuring the free flow of start,” (2021). Available at https://bit.ly/3wNXnVK. materials around the world and continuing 3. UNAIDS, “Statement from the Executive Director of PHARMACEUTICAL the research effort.” UNAIDS, Winnie Byanyima on the decision by the JOINT WITH CONFERENCE United States of America to support the TRIPS waiver LYOPHILIZATION PhRMA (7): “In the midst of a deadly for COVID-19 vaccines,” (2021). Available at https:// pandemic, the Biden Administration has bit.ly/3uNzgp3. taken an unprecedented step that will 4. World Trade Organization, “Waiver From Certain undermine our global response to the Provisions Of The Trips Agreement For The pandemic and compromise safety. This Prevention, Containment And Treatment Of decision will sow confusion between Covid-19 – Responses To Questions,” (2021). public and private partners, further weaken Available at https://bit.ly/3yRdN1n. already strained supply chains and foster the 5. IFPMA, “IFPMA Statement on WTO TRIPS 14-15 SEP 2021 proliferation of counterfeit vaccines. Intellectual Property Waiver,” (2021). Available at LIVE “This change in longstanding American https://bit.ly/3vIXOR9. policy will not save lives. It also flies in the face 6. EFPIA, “EFPIA statement on IP waiver for INTERACTIVE of President Biden’s stated policy of building COVID-19 vaccines,” (2021). Available at https://bit. ONLINE up American infrastructure and creating ly/34xRhNe. jobs by handing over American innovations 7. PhRMA, “PhRMA Statement on WTO TRIPS to countries looking to undermine our Intellectual Property Waiver,” (2021). Available at leadership in biomedical discovery. This https://onphr.ma/3i9aywG REGISTER NOW!
2021 BioManu_TMM_1-3_vert.indd 1 27.05.21 15:15 Delivering the first true innovation since active ® containers. Envirotainer asked hundreds of experts in the air freight industry about the challenges they need to solve. Inspired by their Releye responses and our own 35 years of industry expertise, the team designed the first true innovation since active containers, the Releye® RLP. RLP www.envirotainer.com/releye
100CONTROL MONITORING360 AUTONOMY170 VALUE SUSTAINABILITY90 Through fully redundant Introducing a new dimension Break your dependency on the Releye® RLP reliably delivers Reaching company-wide performance, and a of shipment visibility to the human factor. With up to 170 vaccines∞ and pharmaceuticals at carbon neutrality in 2020, we human-error preventive design, temperature-controlled air hours of unattended a new level of value. Oering both have only just begun. Releye® Releye® RLP provides consistent freight industry: real-time operation, and unlimited if increased flexibility and more RLP achieves up to a 90% ® temperature-control in every status, in your hands, charged, Releye caters to ecient use of the available reduction in CO2 emissions type of situation such as wherever you or your most of all instances of air volume, airline cargo holds can compared to passive solutions. temperature extremes or shipment may be in transportation with zero now be used optimally, adding unplanned door openings. the world. human intervention. invaluable cost-eciency. Delivering the first true innovation since active ® containers. Envirotainer asked hundreds of experts in the air freight industry about the challenges they need to solve. Inspired by their Releye responses and our own 35 years of industry expertise, the team designed the first true innovation since active containers, the Releye® RLP. RLP www.envirotainer.com/releye
100CONTROL MONITORING360 AUTONOMY170 VALUE SUSTAINABILITY90 Through fully redundant Introducing a new dimension Break your dependency on the Releye® RLP reliably delivers Reaching company-wide performance, and a of shipment visibility to the human factor. With up to 170 vaccines∞ and pharmaceuticals at carbon neutrality in 2020, we human-error preventive design, temperature-controlled air hours of unattended a new level of value. Oering both have only just begun. Releye® Releye® RLP provides consistent freight industry: real-time operation, and unlimited if increased flexibility and more RLP achieves up to a 90% ® temperature-control in every status, in your hands, charged, Releye caters to ecient use of the available reduction in CO2 emissions type of situation such as wherever you or your most of all instances of air volume, airline cargo holds can compared to passive solutions. temperature extremes or shipment may be in transportation with zero now be used optimally, adding unplanned door openings. the world. human intervention. invaluable cost-eciency. 14 In My View In My View Experts from across the Navigating the world share a single strongly held opinion Uncertainty or key idea.
How collaboration and innovation – bolstered during the COVID-19 pandemic – will guide the future of stem cell transplantation
By Lori Muffly, Hematologist, Stanford Health Care; Assistant Professor of Medicine (Blood and Marrow Transplantation) at Stanford University Medical Center, Stanford, California, USA
More than a year has passed since the start of the COVID-19 pandemic, marking an appropriate milestone to reflect on how the healthcare system has evolved, the difficulties we have faced, and what we have accomplished for patients during this challenging time. For our team of hematology and blood and marrow transplant specialists at Stanford Health Care, the past year has been full of obstacles but – importantly – also innovative solutions. At the start of the pandemic, we made a crucial decision to continue bringing blood and marrow transplantation (BMT) to patients during COVID-19 coordinators Jennifer McAtee and Leah The current pandemic has made the as long as we could do it safely. Over Schroer, who work tirelessly every day to importance of that network even clearer. the past year, we’ve learned a great deal. oversee and manage the intricate details As new challenges – including a >70 The pandemic forced us to quickly adapt of patient case progression, one of our percent reduction in commercial flights our practices, which has made us more strongest collaborators is the National efficient in ways that we anticipate will Marrow Donor Program (NMDP)/Be continue to save lives. The Match – a nonprofit organization that For the past six years, I have worked manages the collection and transport of “The past year has at Stanford as a physician specializing in donor cells through a worldwide network BMT. Though I am one of the attending of affiliated organizations. From early- been full of obstacles physicians on our team, the full experience morning emergency meetings to late- of transplanting a patient often requires night texts with Jenn and Leah to confirm but – importantly experts who identify suitable donors, a successful match, my work is part of a address financial and logistical roadblocks delicate process enabled by a collaborative – also innovative and, in general, make the work that I do network of people all working toward the possible. In addition to donor search same goal: saving lives. solutions.” In My View 15
(rendering cell transport extremely Services program, which provides pandemic, their advancement during this difficult) and restrictions on in-person financial and logistical support for challenging time was critical. Through donor and patient appointments – were donors within families (reducing the support of our program’s stellar introduced in spring 2020, our team or eliminating the need for family team and our partnership with NMDP/ remained focused on our commitment members to travel). Importantly, the Be The Match, we not only managed to to patients, most of whom were not in a NMDP/Be The Match team developed continue providing transplants during position to wait for a lifesaving transplant. a novel support system to enable related the pandemic, but performed over 400 Making transplantation possible during donor collections during the pandemic, transplants in 2020 – more than any other a pandemic required looking beyond our including custom workup programs to year of our program’s 30+ year history. standard practices. We expedited new ease the process of clearing donors for The way I communicate with patients initiatives to streamline donor matching transplant centers like ours. All these has evolved, too. Although I have and cell collection, overcome logistical changes provide efficiencies that extend always prioritized conversations that barriers, and prioritize donor and patient beyond the pandemic. allow my patients to feel comfortable, safety. NMDP/Be The Match provided the increased uncertainty introduced essential support within each of these areas. by COVID-19 has significantly For example, we knew we needed to underscored the benefits of transparent, expedite HLA confirmatory typing to continuous communication throughout quickly match donors to patients and “I like to tell my the transplant process. As an avid skier, speed the path to transplant. Using I like to tell my patients that navigating NMDP/Be The Match’s FastTrack patients that the steps leading to transplantation is Testing service allowed us to do that. like skiing the bumps – you have to Our team helped NMDP/Be the Match navigating the be nimble and flexible throughout the pilot the FastTrack program prior to the process. As the pandemic has evolved, start of the COVID-19 pandemic – and steps leading to the team and I learned to anticipate the benefits of that early partnership were problems sooner and coach patients greater than we could have imagined. transplantation is more smoothly through the bumps, Another change made by NMDP/ helping them overcome the uncertainty Be the Match in response to travel like skiing the by emphasizing the support systems in uncertainty was to collect donor cells place to guide them. farther in advance of a patient’s transplant bumps – you have COVID-19 has introduced an therapy and to have transplant centers unprecedented level of uncertainty cryopreserve donor cells (i.e., freezing to be nimble and and challenge to the transplant field, cells rather than delivering them fresh) to but it has also led to important positive ensure that they’re available on time for flexible throughout outcomes for researchers, physicians, patients. This provided certainty but also and patients. Our collaborative work introduced new logistical challenges, the process.” with NMDP/Be The Match has because some of Stanford’s cutting- helped us advance the field of stem edge cell therapy research requires the cell transplant in ways that will have use of fresh, unfrozen cells. We carefully an impact far beyond COVID-19. reviewed each patient’s case to determine We also worked closely with NMDP/ Collectively, we are stronger and who qualified for these clinical trials and Be The Match to advance their donor smarter than we were a year ago – and, whether their donor was close enough to optimization efforts, which included through every change, our focus on our center to avoid courier travel delays, the development of a Donor Readiness patients has endured. As our knowledge ensuring that important cancer research Score to calculate key components of evolves, we will continue to prioritize could continue despite the pandemic. donor matching and identify the best sharing our insights with the broader Our BMT program also chose to candidates as quickly and smoothly transplant community and encouraging use in-home and remote typing for as possible. others to share their own experiences our related donor transplants through Though the relevance of these as we create space for continued NMDP/Be The Match’s Related Donor initiatives goes beyond the current innovation in the years to come.
www.themedicinemaker.com 16 In My View
pandemic, the clinical development of Making Good this type of vaccine has been accelerated in an unprecedented manner. There are “Recent Vaccines Better no other examples in drug development where approval has been gained in developments are Mighty mRNA molecules have such a short time. It is also noteworthy won the COVID-19 vaccine that, by working together, individual making it possible race, but there is room for companies and regulatory agencies have improvement when it comes defined a pathway to authorization and for nanoparticulate to effectiveness, if not efficacy delivered the necessary data to allow expedited review. It will be interesting silica to be re- to observe in the coming years if such a process can be applied to more engineered to bind conventional pharmaceutical products. Efficacy of nucleic acids for oligonucleotides of COVID-19 vaccines has now been proven, but effectiveness is becoming varying sizes, more critical as we plan the future management of this disease. including DNA, Demonstrating effectiveness needs to focus on: RNA and SiRNA, • Ensuring vaccines can be easily to the functionalized stored, transported, and used • Ensuring production can be scaled surface of the up to the volumes required in a pandemic in an efficient and cost- particle.” effective way By Nigel Theobald, CEO at N4 Pharma, • Further optimization of delivery UK systems to increase safe release of available to alleviate the potential for nucleic acids into target cells. shortage in any one key component/ Now that several vaccines have reached technology. For example, it has been the market and many others are in A key feature of a good vaccination reported that lipid nanoparticle (LNP) development, I feel it’s a good time strategy, whether in response to a supply is restricted due to shortage in to take stock of where we are – and, pandemic or to endemic disease, is both lipid components and assembly more importantly, where we are going. to have multiple versions/types of technology at scale. And I think it is also important to vaccine readily available that can When it comes to delivery systems make the distinction between efficacy respond quickly to natural mutation or strategies, the new COVID-19 (reduction of disease) and effectiveness in the virus. Current sequencing vaccines rely on either LNPs, which, (ability to achieve immunity in the mass technology allows ready identification as noted above, may be subject to supply population) of the vaccines that are of novel variants, which may alter the constraints, as well as having drawbacks available and being developed. We must binding characteristics of, for example, in terms of possible toxicity and sub- also consider what lessons can be learnt the SARS-CoV-2 Spike protein. By optimal cellular penetration; or viral as we plan the future management using that sequence, mRNA can be vectors, which can cause side-effects. of this (and other) contagious constructed and delivered to pharma However, silica nanoparticles could disease outbreaks. manufacturers for incorporation into help to overcome some of these issues. The potential for DNA/RNA their proprietary delivery systems. Silica is “Generally Recognized As vaccines has been explored for many An equally important point is that Safe” by the FDA and mesoporous silica years. Out of necessity, and early in the several delivery systems should be nanoparticles (MSNs) – silica-based In My View 17
nanostructured materials with strong oligonucleotides of varying sizes, other morphologies and a commercial biocompatibility and chemical stability including DNA, RNA and SiRNA, transfection agent. Out of the three, – have been used in many successful to the functionalized surface of the the spiky nanoparticles were shown drug formulations already. They do, particle. By functionalizing silica to to best facilitate efficient cellular however, have significant limitations alter its topography, researchers are now uptake, endosomal escape and delivery for nucleic acid delivery. While inert demonstrating how it can be considered of pDNA to the nucleus, leading to and safe, most silica systems tested to a viable delivery system for nucleic successful intracellular gene expression date have been smooth mesoporous acids. In a comparative study, scientists and the highest transfection rate. particles, meaning the nucleic acid is at the University of Queensland (UQ ) Given initial findings into the attached to the side of the particle, investigated how the topography of ability of silica nanoparticles to limiting the amount that would be SNPs impacts their performance as a also offer enzymatic protection successfully delivered into the cell. An nucleic acid delivery system (1). SNPs to oligonucleotides and provide alternative, effective, non-lipid delivery with spiky-, raspberry- and flower- formulation stability, I believe there is solution that protects the nucleic acid, like morphologies were constructed a strong role for silica to play in the delivers enough of it into the cell for with spike, hemisphere and bowl type vaccine field over the next five years. the required immune response, and subunits, respectively. Scientists at UQ ensures safety and immunogenicity found that the spiky-type subunits Reference was, therefore, required. exhibited stronger binding affinity 1. H Song et al., “Plasmid DNA Delivery Recent developments are making towards pDNA molecules and allowed Nanotopography Matters,” Journal of the it possible for nanoparticulate effective protection against nuclease American Chemical Society, 139, 18247 silica to be re-engineered to bind degradation when compared with the (2017). DOI: 10.1021/jacs.7b08974.
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teams away from the vital work that Powering drives innovative new treatments and therapies. Digitalization can optimize “Lack of real-time Discovery with these non-core tasks and give scientists more time for discovery. visibility into basic Digitalization Lack of real-time visibility into basic resources is a core challenge for many resources is a Inventory management tools, labs. From the number of centrifuges on Internet of Things, automated the shelf to whether equipment should core challenge for replenishment, AI… there are be repaired or discarded, labs without myriad opportunities to use accurate, instantaneous data must many labs.” digital tech to improve lab often wait weeks or months to identify efficiency trends, react to evolving situations, or make decisions. analytics that are based on past trends Though many labs have already taken and habits. steps toward digitalization to operate Digitalization can also significantly more efficiently and prevent costly enhance transparency into laboratory downtime, the COVID-19 pandemic chemical management. Often, further highlighted the need for real- overstocked inventory, lost chemicals, time information. As labs sought to and potentially expired substances quickly develop safe and effective are not uncovered until an audit is treatments and vaccines, they needed performed. Digitalization tools can help visibility into their processes and reduce this waste. For example, RFID workflows. Organizations that already can help locate and monitor chemical had robust digital solutions were better stock and provide real-time usage data. positioned to tap into the transparency This allows laboratories to monitor needed to quickly pivot in response chemicals, ensure proper handling and to global events. As we move into a storage, and help determine the proper post-COVID landscape, it will be time for disposal and replacement. more critical than ever for labs to have Laboratories can also use actionable intelligence that helps them digitalization tools to manage get treatments to market faster. equipment over its full life cycle. Lack In my view, lab digitalization of visibility into equipment, from flasks can help manage a wide range of to freezers, can lead to unplanned needs, from day-to-day operations to downtime, research delays, and regulatory compliance. For example, noncompliant documentation. Labs can inventory management tools such as more efficiently manage the complexity By Tola Olorunnisola, President of smart shelves and point-of-use sensors of equipment lifecycle management Innovation, Marketing and Digital at bring Internet of Things (IoT)-based with tools such as a digital repository Avantor, Radnor, Pennsylvania, USA technology into the lab by providing that makes it easy to record audits or real-time usage information and real-time data that indicates it is time to Scientists are on a mission to make automated replenishment. This reduces decommission. Likewise, digitalization the world a better place. But to drive the number of human interventions can help labs navigate the evolving discovery and innovation, companies using sensor-triggered demand signals clinical trials landscape. As clinical must maximize laboratory productivity instead of physical time interval trial sponsors increasingly embrace in an increasingly costly – and fast- checks. In addition, moving from lab global multisite trials and decentralized paced – research environment. automation to analyzing data on what trials, the implementation of sample Though non-core scientific tasks such scientists are doing and which protocols management and technology, such as as lab operations are critical, they take they are working on produces predictive RFID-based tracking and tracing, can In My View 19
help researchers ensure supply chain for chromatography, researchers need if available and identifying what other transparency and trial integrity. information about the molecules, the condition settings are required to Although knowing what will conditions, and the resins. As more data optimize the experiment. happen, when it will happen, and why points are connected, AI technology To get treatments to market is valuable, companies can further can analyze the data and make faster, laboratories must maximize leverage digitalization capabilities to recommendations for more effective, productivity. Whether a lab manages move into prescriptive analytics that more environmentally friendly solvents digitalization in-house or works with make recommendations and indicate – and, most importantly, provide an outsourcing services partner, these their potential outcomes. This can be information on any parameters that advanced technologies can provide the done by connecting the right systems might need to be adjusted. In another visibility researchers need to optimize and tapping into existing data. For example, this technology could the lab environment so they can focus example, in a laboratory that uses analyze a cell culture growing in a on what matters most: discovery methylene chloride (HPLC) solvent flask, suggesting better flask options and innovation.
decision-makers to carefully consider Roadmapping their technology selections with the entire potential product lifecycle in Future mind. Aspects under consideration might include scale-up and scale-down Technology performance, the physical operations and space required, the number of How do we mitigate the processes to which the technology risks and costs of choosing can be applied, and how the selection inappropriate technologies for might limit or expand future processing the short and long term? platforms. One might also consider market aspects, such as assurance By Mark Thomas Smith, Staff Engineer of supply and delivery lead times, in Bioprocess Technology Development at possible alternative and replacement Thermo Fisher Scientific, Logan, technologies and, perhaps most Utah, USA important, the costs of implementing those technologies at some later date, Bioproduction – the process of if needed. manufacturing biopharmaceuticals or their precursors – has experienced product development lifecycle and persistent, yet discontinuous can take months or even years to be technological progress ever since implemented for manufacturing’s “New filtration the release of the first blockbuster benefit. Even in the earliest stages of a biologics. Major technological advances new product development cycle, there modalities, such as have taken place in various aspects of is a propensity to design bioprocesses bioproduction, from cell biology and strictly on well-known technologies, tangential flow gene integration to bioprocess media which is sometimes heralded as a and chromatographic technologies. “platform process” – in a sense, “if it filtration, However, due to regulatory, cost, ain’t broke, don’t fix it.” and other risk factors, it can be With such potential difficulties have been (re) difficult to implement new bioprocess integrating new bioproduction technologies. New technologies are technologies into existing processes, introduced.” typically introduced early in a new it behooves development teams and
www.themedicinemaker.com 20 In My View
Let us consider the narrow example product yields. Alternatively, new effect in which its perceived value and of selecting technologies for cell culture filtration modalities, such as tangential ease are exaggerated and the relative harvest and clarification. Traditionally, flow filtration, have been (re)introduced. value and ease of unknown technologies for vessels above 10,000 L, harvest was These have considerable implications are downplayed. performed with stainless-steel disc in the residual of the bioprocess, How do we, as an industry, mitigate stack centrifuges and post-centrifugal potentially requiring development the risks and costs of choosing depth filters (1). As cell biology work across multiple bioprocess unit inappropriate technologies for the and culture technologies improved, operations (e.g., moving to perfusion- short and long term without exhaustive, product titers also improved. Increased based upstream). These and other expensive research efforts? One avenue titers, combined with flexibility and technologies are available, but each one is through industrial forums, such as regulatory considerations, helped drive comes with additional risks, challenges, the BioPhorum Group (BPOG), the a shift in bioprocesses from large CIP/ or burdens. These would become less International Society of Pharmaceutical SIP stainless steel to smaller single-use daunting if the technology’s necessity Engineers (ISPE), and the Bio-Process Optimize every lab. equipment, particularly for bioreactors. was realized from the start. System Alliance (BPSA). Technology At smaller volumes and moderate roadmapping plays a heavy part in these titers and cell densities, the stainless forums, helping end-users to identify steel centrifuges could be replaced future technological bottlenecks and Every way. Every day. with single-use depth filters, reducing “How do we, as an allowing vendors to collaboratively the facility engineering capital and tune in. Overall, a level of transparency operations. industry, mitigate and communication between vendors But adopters of single-use depth and users is established, creating a filtration now face a rising challenge: the risks and costs more efficient marketplace for new upstream culture technologies have technologies to arise in a timely fashion. Maximize lab efficiency with scalable, continued to intensify and single-use of choosing But having technologies is not the reactors are available in larger sizes same as implementing technologies. end-to-end solutions from Avantor® Services. (e.g., 5,000 L). These improvements inappropriate Beyond roadmapping needed portend the end of some large stainless technologies, it is also critical to steel bioreactors, promising to be more technologies for the collaboratively consider other hurdles. Scientific discovery is time-consuming and resource-intensive. Efficiency and speed to market is critical in flexible and cost-effective than massive In particular, the obstacles to regulatory advancing your scientific efforts. You need a partner you can trust to deliver scalable solutions to help optimize stainless steel vessels for multi-product short and long term implementation need to be addressed. By your lab operations and advance your research. facilities addressing smaller indication working hand-in-hand, vendors, users, populations. However, the cell culture without and regulators can leverage forums From one-stop procurement to inventory management to technical lab support, Avantor Services offers the volumes and intensities are beginning and other collaborations to establish deep expertise and global solutions you need to streamline processes in every workflow, create peak efficiencies, to stretch the capabilities of single-use exhaustive, best practices that satisfy regulations eliminate bottlenecks and free high-value resources through: depth filtration to the edge of being while shaping the future of regulatory — Lab & production services with on- and off- — Digital solutions including hardware, software and tenable in performance, economics, requirements. If we can become more expensive research site support for general and technical lab advanced analysis solutions for every stage of lab or both. efficient, informed, and decisive about services, procurement, production and business management Although some processes are locked efforts?” planning and implementing the right process consulting into traditional single-use depth new technology today as we look toward — Clinical services with a team of experts delivering filters, there are alternatives available the future, the industry, the product, and — Equipment services offering full equipment custom kitting service, biorepository and archiving at varying levels of maturity and – most importantly – the patient will be life cycle management from identification to services, and equipment and ancillary solutions, impact. For example, several companies This brief example of selecting harvest better for it. decommissioning, including multivendor technical anywhere in the world services and certification have developed filters to be paired technology, though trite, highlights that — Commercial kitting & services providing global with flocculants, enabling harvest there are numerous risks and challenges Reference — Procurement and sourcing services providing expertise, logistical support and scalable kitting of relatively high-density cultures; a in adopting new technologies. Effort 1. AW Dryden et al., “Technical and economic simplified, one-stop procurement process, expert solutions potential tradeoff is that the addition should be made at the time of initial considerations of cell culture harvest and product advice and responsive fulfillment of flocculants may cause additional selection to consider the long-term clarification technologies,” Biochem Eng J, purification and development burdens, ramifications. A technology, once 167 (2021). DOI: 10.1016/j. as well as potentially result in lower known, is subject to an endowment bej.2020.107892. Move research and discovery forward with Avantor avantorsciences.com/avantor-services Optimize every lab. Every way. Every day.
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Feature 23
How will artificial intelligence help pharmaceutical innovation reach new heights?
By Maryam Mahdi and Stephanie Sutton
is changing the world around us – affecting every aspect of our lives, whetherAI we realize it or not. For (big) pharma, there is the opportunity to use these platforms to streamline many processes, from R&D to manufacturing to business operations. But change is always resisted. And though many companies are happily stepping – or even running – towards an AI-driven future, some are still assessing the risks and full implications of these technologies. The COVID-19 pandemic is clearly acting as a catalyst for change, but will the industry be able to make the most of the advanced – and rapidly advancing – tools at its disposal? In this month’s cover feature, we explore how AI will shape the next era of pharmaceutical research and development.
www.themedicinemaker.com 24 Feature
BEYOND DRUG computing capabilities in just a few days. The ability to harness DEVELOPMENT enormous computing power has had a huge impact on what the industry can now do with AI. AI opens up new opportunities in R&D – and also The COVID Moonshot has been exciting for all concerned. provides pharma companies with new ways to connect They have the output from 2020 and are approaching the with patients for better outcomes candidate stage. And I really hope we see some great results. For UCB, the COVID Moonshot has helped us see directly how rapid Combining cutting-edge computing power and AI access to large-scale computing capabilities can really transform algorithms can significantly accelerate drug discovery by medicines discovery. UCB is not a leader in infectious disease giving scientists a vast chemical space to test hypotheses and R&D, but the same approach can help us speed up discovery identify more effective molecules. Roger Palframan, Head stage programs – regardless of therapeutic area. And we have External Innovation and US Discovery at UCB, tells us how plans to deploy the technology on a number of interesting projects. AI is making a difference to both the company and patients. How did you get involved with Microsoft? Why is artificial intelligence so important Like many companies, we had a long-standing relationship with for the pharma industry? Microsoft at the enterprise level. When we decided we wanted to At UCB, we see artificial intelligence as one type of enabling work on the COVID Moonshot, we approached them through advanced analytics tool that helps our people make better – their “AI for Health” program to access the cloud platform. We’ve and faster – decisions. It can benefit research scientists, clinical now expanded our collaboration with Microsoft to combine their scientists, and other different parts of the business. computational services, cloud, and AI with our drug discovery We tend to talk about AI in three different areas. First, we and development capabilities. A lot of drug discovery activities use it to understand the patient and their condition as deeply require the analysis of high-dimensional data sets or multi-modal as possible; how does the disease impact the patient population unstructured information; here, computing power is crucial. and individual patients? It’s important to understand the unmet We’re also going to be using a new Microsoft platform to deploy need so we can really create value for patients. And that includes AI across the enterprise. gaining a better understanding of pathophysiology. This takes us to the second area that is the search for better targets; we want How have UCB scientists reacted to AI to increase the probability that, by perturbing or modulating a technology? certain target, we can improve the right clinical outcomes for Many people in the company were already using AI before the patient. And the third area is all about getting to the right we increased our focus in this area; our data scientists and therapeutic candidates faster, whether a small molecule, biologic, computational chemists were already using AI day to day, and we or gene therapy. We want approaches that can accelerate decision also use AI in other parts of the business, including non-scientific making, help us prioritize, and shorten our to-do list on the road roles and support functions. But we really want to see AI in the to potential therapeutics! hands of as many scientists as possible, and I think that’s what the Microsoft collaboration can really enable us to do. When did UCB first become interested in AI? For our scientists, AI has had a huge impact in drug discovery. I wouldn’t say there was a specific time UCB pivoted towards We have programs where a target was previously thought to be AI. Over time, there has been a convergence of computing undruggable, but we have used computational design approaches power and data availability that has significantly accelerated its to find a way forward by identifying molecules that can be adoption. Many of the AI algorithms are not new; rather, we combined to modulate the function of a particular protein. In needed a way to make the data ready at scale for analysis using fact, there are many exciting targets with really strong causal these algorithms, as well as the necessary computing power to links to disease that have proved to be very difficult to address, perform the analytics process. And it all had to be affordable. The particularly with small molecule drugs. convergence of these factors has been very important and has led As another example (which isn’t strictly AI, but probably would to projects like the COVID Moonshot crowdsourcing initiative. be seen as a type of AI today): our investigational humanized We brought algorithms and approaches to the Moonshot that we monoclonal IgG1 antibody bimekizumab was optimized to had already developed in our computer-aided drug design group. selectively and directly inhibit both IL-17A and IL-17F, two Our approaches make use of the Microsoft Azure cloud, which key cytokines driving inflammatory processes, by superimposing allows us to perform tasks that would take months with UCB’s two binding specificities into a single binding site. This advance C O V I D M O O N S H O T
The COVID Moonshot is an open group of researchers – including biopharma, academia, technology companies, chemical vendors, and individuals – who are working under no IP constraints on new compounds and solutions to tackle COVID-19. The initiative has crowdsourced over 17,000 chemical designs for antivirals from chemists around the world. Volunteers can also help by sharing their computer power for molecular simulations. M E E T R O G E R The project is led by PostEra, PALFRAMAN which specializes in medicinal chemistry and machine learning. My background is in life science, pharmacology, and UCB has contributed employee time immunology. I did my undergraduate studies at King’s in the areas of medicinal chemistry, College London, and PhD at Imperial College London, CADD, and IT to contribute before doing a postdoctoral research fellowship, under drug design ideas and prioritize the Wellcome Trust, at Harvard Medical School. My crowdsourced submissions (more move to industry was spurred by my desire to apply than 13,000 so far). science to therapeutics discovery. I joined Celltech, which became part of UCB in 2004, and I’ve had a number of Find out more at opportunities to gain experience since – leading groups https://covid.postera.ai/covid in discovery research, working on significant clinical development programs, and feeding into strategy. In 2015, I came to Boston to enable UCB to tap into the life sciences hub here and across the US. We’ve always had a presence in the US, but not in terms of research – despite was made possible through proprietary computational antibody collaborations with leading universities and biopharma. We design – and we demonstrated that the result had superior clinical started in an office at the Cambridge Innovation Center and benefit to IL-17A-only antibodies. Such discovery capability is we’ve now established our own research capabilities in the core to what we do at UCB. US – started by the acquisition of Beryllium Discovery and We are also using AI to better understand disease, because it’s Element Genomics – and accelerated with the acquisition very good at pattern recognition. We are collaborating with an of Ra Pharmaceuticals in Cambridge in 2020. academic partner to help us identify subpopulations in epilepsy. We’ve used AI to analyze over 100 million closed datasets alongside a smaller subset (around three million) of detailed electronic medical records. And we’ve identified 40 previously is something we are very passionate about. We recently uncategorized subpopulations with epilepsy. We’re considered a announced the launch of Nile AI – an independent digital leader in the epilepsy field so it’s important for us to understand health platform that improves the delivery of epilepsy care these phenotypic clusters so that we can better help those patients by shortening the path to optimal treatment. The platform get the care they need, either with existing therapeutics or with consists of a patient app and healthcare provider portal. The new therapeutics that we have yet to discover. portal shows the status of patients at a glance, and virtually informs and supports patients in between appointments, and, How do you think AI will shape the future crucially, it also allows data-driven care-related decisions. We of healthcare? hope it will benefit both patients and healthcare providers. One of the biggest challenges in healthcare overall is allowing Being a pharma company isn’t just about developing patients to access the right medicine as quickly and effortlessly therapeutics. It’s important to be working on solutions that as possible. Using AI and digital technologies to help patients help the patient holistically.
www.themedicinemaker.com 26 Feature
A MORE INTELLIGENT successful AI implementation. There is no room for error when APPROACH it comes to these systems, and we need specialist data experts to collaborate with scientists to meet this particular challenge. Has AI been a savior in the battle against Another barrier is the lack of clear data standards. When COVID-19? data is disorganized and siloed it is not machine-readable, which has the potential to create bias in AI’s outputs; in other AI has helped inform pharma’s COVID-19 response. But words, the information that it is using to “train” its algorithm how? We take a deep dive into the industry’s growing reliance is limited. on AI systems by speaking with The Pistoia Alliance President, The Pistoia Alliance has developed a toolkit called FAIR Steve Arlington. (Findable, Accessible, Interoperable, and Reusable) that aims to address this algorithmic bias when companies are setting What challenges have been highlighted by up new data sets (See Technical Support). The resources are the pandemic? free for companies to access and should enable a smoother The pandemic has exposed long-standing industry issues, transition towards increased use of AI systems. including difficulties accessing data outside of the lab and the lack of ways for organizations to collaborate virtually And what about cybersecurity? both internally and externally. These problems have the As with any new technology, security is an important potential to lead to duplication of work. And with factor but arguably even more so when dealing more scientists working remotely than ever with sensitive patient data. Ensuring AI is before, the pandemic has shown us the explainable and removing the “black box” true value of digital tools in helping can help to diminish security concerns. overcome these problems. The search When AI is transparent and the for COVID-19 treatments has also source of the data (and decisions reiterated the vast amount and “We hope this security it makes) can be easily traced variety of data produced by the advantage can improve and understood, it is easier for pharmaceutical industry as well as patient trust and encourage tampering to be uncovered. Other the need for technology that makes more knowledge sharing technologies, such as blockchain, the management and sharing of this across the life science also have the potential to increase data more straightforward. The last security because of its trusted, year has proven that collaboration community.” disintermediated system of record. leads to faster breakthroughs; we must We hope this security advantage can continue to cultivate this cooperative improve patient trust and encourage attitude to research, and support scientists more knowledge sharing across the life by implementing the right technologies. science community. Any emerging tech still requires us to work What role can AI play in addressing together to put the right standards, governance, and these issues? guidelines in place to minimize security risks. AI can aid the management and sharing of scientific data. It improves the success rates of drug discovery by sifting How can pharma prepare itself for future through huge existing data sets and using them to answer pandemics? new questions – as we saw with the rapid development COVID-19 presented the life sciences with a complex of COVID-19 vaccines. AI can also make searching challenge, but it has also been a catalyst for biopharma for and finding insights from data much more efficient, organizations to work together in new ways with technology letting researchers use existing knowledge and avoid companies, governments, charities, and regulators. From overlapping results. repurposed drugs to new mRNA vaccines, R&D has been accelerated during the pandemic. Technologies such as AI Why are some companies still hesitant to have facilitated this, but ultimately it is collaboration that adopt AI? will drive scientific breakthroughs during any future public The skills gap is the biggest recurring barrier preventing health threats. T E C H N I C A L to help companies find, use (and suggest model management for SUPPORT reuse) the data most relevant to both AI and machine learning. It them in an ever-growing virtual sea also regularly hosts webinars to The Pistoia Alliance is involved of information. Now the project helps better acquaint its members with the in projects that allow industry companies gain access to free tools, changing AI landscape. The Alliance partners to share best practices training, and change management also encourages international and discuss ways of overcoming programs. Over 50 data experts and collaboration through the center. common challenges experienced in 18 companies are associated with Its virtual co-working spaces allow the use of AI. FAIR – helping to enhance clinical companies across different regions trial data management for future to share ideas and collaborate on the FAIR Toolkit drug development. implementation of AI within their Motivated by the industry’s organizational structures. increasing reliance on data-driven AI Centre of Excellence solutions, the Pistoia Alliance set (CoE) Through the AI CoE, they have also up FAIR. The project, which first The Pistoia Alliance launched its published a list of best practices that began in 2016, published its first CoE in 2017. The project aims to can be found online set of guidelines in Scientific Data explore the ethics of AI use and https://osf.io/eqm9j.
T H E D R E A M treatments. Some of their better-known immunotherapy datasets and ultimately W O R K S collaborators include Pfizer, Boehringer offers the company the ability to Ingelheim, and Actoris. efficiently explore clinical data insights. Perspectives on AI are rapidly Microsoft is also working closely with changing. In 2015, there Exscientia big pharma companies. For example, it were only 20 collaborations Exscientia is also turning heads in the recently signed a multi-year deal in 2019 between pharmaceutical pharmaceutical sector. The AI company that enabled Novartis to set up its AI companies and AI startups. recently extended its Bristol-Myers- innovation lab to support staff in the drug Today, the landscape looks very Squibb partnership with a view to expand development process. In a statement, different. But what impact are its portfolio of small molecule drugs Novartis’ CEO Vas Narasimhan said, these smaller tech companies for a variety of indications including “As Novartis continues evolving into a having on drug discovery and oncology and immunology. “Exscientia’s focused medicines company powered development? Here, we round application of AI technologies is proving by advanced therapy platforms and data up just a snapshot of the projects capable of reducing discovery times. science, alliances like this will help us underway. Rapid discovery of molecules that can deliver on our purpose to reimagine enter the clinic in a timely manner could medicine to improve and extend lives.” Insilico Medicine positively impact our work in discovering Insilico Medicine has made huge strides treatments for areas of unmet medical BenevolentAI in the use of AI for drug development in need,” said Rupert Vessey, President In January, AstraZeneca announced that recent months. Using its AI platform, of Research & Early Development at it had partnered with BenevolentAI to the company discovered and developed Bristol Myers Squibb in a statement. launch its first AI-generated target for a drug to treat idiopathic pulmonary chronic kidney disease. However, this fibrosis in the space of 18 months. Microsoft isn’t the tech company’s first foray into Their work has helped demonstrate The tech giant is playing a large role big pharma; they joined forces with the power of AI for rapid R&D and in the drug development space, using Novartis almost two years ago to support has attracted several partners who its AI prowess to support a variety of its oncology program. The deal saw are using their proprietary platform, companies with its proprietary cloud- BenevolentAI using its deep learning Pandomics Discovery, for its ability to based computing platform, Azure. In platform to screen Novartis’ current provide relevant real-world evidence the world of pharma, Azure allows for pipeline to identify drugs that could be for the development of new drugs and the storage and streamlining of large repurposed for other indications.
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CULTURE SHOCK provide critical execution support. Leveraging a modular AI approach, commercial teams can easily configure a right-size Do pharma organizations have the right mindset when solution for quickly identifying the right commercial targets, it comes to adopting and implementing AI more widely? predicting the ideal timing and content for maximum HCP engagement, and prioritizing next best actions that users are By Alan Kalton most likely to adopt. There are several emerging biotechs that are leveraging AI is constantly evolving. In the early 1990s, basic AI systems AI in really fundamentally new ways – and that is largely were used to apply best practice logic to help track complex because they are not stuck in the quicksand of investment that scenarios. Now, AI systems are more powerful than ever and large companies have made in their field forces, marketing can be used in various ways across the life sciences industry. automation systems, processes, and more. They can start In addition to drug discovery, pharmaceutical companies can fresh. One customer is building its commercial team anew leverage AI to explore the major trends that drive performance using AI as the engine to power and coordinate a digital-only and affect sales and marketing, as well as the minute details engagement model. They are essentially ‘bucking the system’ of consumer behavior to help improve the patient experience. of investing first in the field organization. Post COVID-19, it’s Most commonly, AI uncovers customer preferences – such as like a phoenix rising from the ashes and zeroing in on digital preferred channel, preferred time to engage, preferred type of engagement directed by AI intelligence. information – so commercial teams can enhance engagement Another customer is building its commercial team based on with healthcare professionals (HCPs). Further, AI quickly data – a total break from tradition. The company is investing derives insights from mountains of data to guide marketing in data technology that integrates all engagement data into a teams in developing materials that better meet the specific consistent framework as a baseline for driving an AI-first agenda needs of HCPs and their patients. AI allows personalized and data-driven approach to commercialization. This move will interactions like never before – it replaces mass marketing accelerate their AI journey faster than other companies. with tailored, relevant communications. But a challenge for any business when initially considering Although the uptake of AI is on the rise, management AI implementation is what AI truly means and how it can must shift its mindset to ensure that the systems are used positively shape their futures. efficiently. In my experience, large companies often have different attitudes towards these systems versus small and Understanding AI medium-sized companies. Although large companies will have You can ask 20 different sales or marketing professionals from the capital and resources to pursue ambitious projects, they the industry what AI is – and receive as many answers. The can sometimes be entrenched in decades of protocol, process, fact of the matter is that for pharma to agree on a consistent and tradition. Therefore, understanding how AI can best be definition, the industry as a whole must be able to identify the implemented – and rolling it out – can seem daunting. The types of problems it is trying to solve using these AI platforms. functional silos that often exist in these organizations can For example, when extracting relevant information from large hinder the use of AI at scale. Without the ability to make datasets, I’ve heard the terms “multichannel” (an information- effective decisions across departments, progress can be slow centered approach), “omnichannel” (customer-centered and the success both locally and internationally can be affected. approach), and “digital” all used interchangeably within the In contrast, the smaller, more agile companies are leading the same company to refer to the analysis of the same products with charge and engaging with AI. They are willing to explore how AI. No wonder there is so much confusion about AI! products can be launched within an entirely digital framework There is also a lack of real-world and direct experience with and how AI can impact commercial success worldwide. They AI for professionals and their companies to draw from and ask tough questions to ensure that the structure of their build upon. This is, in part, due to the closed nature of the processes benefit their operations and their investments are pharmaceutical industry – although professionals will move to effectively used. and impart their knowledge on different companies within the When COVID-19 hit, these rapidly growing biopharma sector, the voices of skilled individuals from other industries companies had to abruptly adapt to a new commercial are often missing from the equation – especially when it comes reality. Antares Pharma, for example, sought innovative to the execution of commercial models. Pharma has tried ways to continue engaging with customers and turned to AI to recruit digital technology innovators from outside of the technology to help bolster internal analytics capabilities and industry but they quickly grow frustrated by the rigidity of the Feature 29
traditional commercial model and unique regulatory processes in pharma. The initiatives that they try to develop fail to go beyond the pilot stage and these leaders move on. Essentially, there is a skills gap when it comes to using AI for intelligent customer engagement in pharma. The good news is that this paradigm is finally starting to change. As the life sciences industry has accepted the need for AI to be more ubiquitous, AI skillsets are in demand and the industry is primed for the requisite changes that must take place. Pharma companies will still need help taking AI innovation to scale but the mindset is beginning to evolve – some might say by necessity because of the lingering impact of COVID-19. AI can act as a catalyst for strategic change (and many companies are introducing it into their practices), but its dynamic nature has yet to be fully explored on a broad “If we look back at scale. Companies should be able to use the data available to them to move the last 18 months, we away from quarterly plans and annual can clearly see how the reviews, and execute strategies on areas pandemic has changed how it can be improved. like patient engagement on an ad hoc attitudes in pharma” Now, the potential for AI beyond basis – potentially as frequently as the transactional excellence is about market changes. Recent events, including driving a dynamic relationship between the COVID-19 pandemic, have further strategy and tactics. For example, emphasized how important it is for the industry AI allows pharmaceutical companies to to consistently and rapidly address issues that can transition away from an annual planning cycle to negatively impact the patient experience. a continuous, in-real-time cycle. Using rules-based AI, companies can quickly pivot to address a significant change The COVID-19 impact such as a new indication for a drug, a new competitive entry, If we look back at the last 18 months, we can clearly see a safety issue, or a regulatory change. This is the holy grail of how the pandemic has changed attitudes in pharma. It has what AI can do for the life sciences industry and represents a brought into question how companies were allocating resources fundamental shift in strategic operations from the executive across the entire commercial spectrum and beyond individual level on down. business concerns; we’ve all had to consider the macroeconomic In short, there is a lot to unpack when it comes to “true consequences that came along with the uncertainty of the transformation” for pharma’s use of AI in customer engagement. crisis. We didn’t have a clear picture of how it would affect Throughout my time spent in industry, I’ve seen the phrase the industry as a whole on a long-term basis. used many times and have also experienced the intransigence These challenges have meant that we’re now seeing greater of the pharmaceutical commercial model. Though pharma levels of innovation in areas of AI use that were previously has had over 100 years of success, it hasn’t fully assessed underfunded, such as for omnichannel engagement in the limitations of current strategic models. COVID-19 has commercial. AI has also transitioned from a toy for companies offered us all a real chance for transformation and is inspiring a to “play” with, to an essential tool as it became increasingly behavioral change in companies of all sizes. It’s phenomenally apparent that we would have to quickly respond to arising exciting. I hope this enthusiasm and shift in mindset won’t be issues and ensure that commercial and strategic models lost; we have now a golden opportunity to connect AI to an allowed us to compete in the marketplace. COVID-19 made improved patient experience. the need for digital and AI’s application to enable smart digital engagement with customers crystal clear. It has also helped Alan Kalton is vice president and general manager, us to think more critically about the customer experience and EMEA at Aktana.
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IS THERE GOLD IN THEM witnessed huge scientific leaps in vaccine development. AI HILLS? Lastly, the unprecedented way in which data has been shared so rapidly and freely during the pandemic has raised important What opportunities await pharma as it begins to questions about what data is proprietary and the possibilities redirect its focus away from the pandemic? of pre-competitive sharing. We will need to see discussions about the standardization of data capture, how we increase In 2020, we spoke to Peter Richardson, Vice President of diversity in data, and ensure patients have greater feedback Pharmacology at BenevolentAI, about the role the company’s on how their data is being used. AI system had played in its pandemic response. Like many other companies in the industry, they put their machine learning platform How can we accelerate drug development to use and found that a drug owned by Eli Lilly, baricitinib, could projects of the future? be repurposed to treat the symptoms of COVID-19. BenevolentAI and other industry pioneers have demonstrated And although AI has played a significant role in the the power of AI, combined with human intelligence, to industry’s rapid drug development program through the generate new hypotheses for serious diseases and identify crisis, stakeholders in the industry are now looking ahead to targets faster and more cost-effectively. However, to change the determine the role it will play in a post-COVID-19 world. To drug discovery and development process worldwide, we need to examine the topic further, we caught up with Jackie Hunter, a see substantial organizational change and significant effort on board director at the company and a member of The Medicine the part of pharma companies. For example, increasing the use Maker 2021 Power List, who shared her views on the future of remote monitoring and standardization of image analysis applications of AI in pharma with us. to reduce the number of patients needed in trials. Industry challenges can also be solved through productive How will industry conversations change in pharma-innovator partnerships, for example, we partnered a post-pandemic environment? with AstraZeneca to uncover novel treatments for idiopathic Last year, the company identified baricitinib as a treatment pulmonary fibrosis and chronic kidney disease. And that led for COVID-19, which later won FDA approval for use in to AstraZeneca selecting the first-ever novel AI-generated hospitalized patients. Post-pandemic, we should expect target to enter its drug portfolio. However, these types of industry conversations to revolve around how we use AI to partnerships are only truly successful if both sides are willing improve future health systems and revolutionize the drug to learn from one another and adapt their ways of working to discovery process. Another topic that will dominate the combine the expertise of traditional pharma with new AI and industry post-COVID is how we improve and unblock machine learning approaches. In the case of Benevolent and approval processes for future therapies and vaccines. We have AstraZeneca, this led to AstraZeneca selecting their first-ever seen how approvals can be expedited with rolling data reviews novel AI-generated target from the partnership to enter their and large data trials in the pandemic, and, as a result, we have drug portfolio. What technologies will drive the future of pharma? Clearly, AI and machine learning will impact every aspect of the pharma value chain, and 2020 saw the field make impressive progress on some of the world’s most complex scientific challenges, such as AlphaFold (a program developed by GoogleAI offshoot Deepmind for accurate 3D predictions on protein folding) or MIT’s work in antibiotic discovery. I think this closer convergence of new technologies with scientific expertise will help the industry be more successful, efficient, and cost-effective as it moves forward. I also hope to see other exciting technologies; for example, blockchain to help patients track how their data is used and smart manufacturing technologies, such as 3D printing, to enable a more personalized approach to medicine. Feature 31
GROWING INTELLIGENCE
Distribution of AI companies for drug discovery by region
US – 54.4% Canada – 6.7% UK – 14.2% EU – 13.4% China – 2.5% Rest of Asia – 8.4% Australia – 0.4%
Is there a strong Can AI help drugs get to Will failure to adopt AI digital culture in market faster and result in financial pharma? eliminate conventional consequences? challenges?
Excellent 34% Yes 50% Yes 43% Adequate 52% No 50% No 57% Poor 14%
2000 Pharma’s Global Investment in AI Sources: 1500 MIT Technology Review (2021). https://bit. 1000 ly/2RJq7QK Deep Pharma 500 Intelligence AI Investment (US$ million) (2021). https://bit. ly/3hNYK2F 0 Aspen Tech 2014 2015 2016 2016 2017 2018 2019 2020 (2021). https://bit. ly/34cO99k. 32 Sponsored Feature
curve too as we adopted more digital and clients who were troubleshooting a process Global Support virtual technologies to meet customers’ issue at their manufacturing site in Asia. Such needs, but we’ve managed to navigate interaction is only possible when you invest for Faster through the situation successfully. in global expertise and digital technology. The Colorcon team are equipped to Formulation Tell us about your US expansion... provide the same level of support wherever CC: We recently extended our technical they are based in the world. Development service capabilities in our global HQ in Harleysville, PA, USA with a new What emerging trends are impacting Driven by innovation and the containment suite. Many of our customers customer’s needs? desire to meet new treatment work with very small quantities of active CC: There is increasing demand for product options, pharmaceutical ingredients, which can have unknown safety authentication driven by the tremendous companies worldwide are looking profiles, and be extremely costly growth of the drug counterfeiting to accelerate time to market and limited in availability. industry; especially with for their products. Experts Our investment in the proliferation of from Colorcon explain how the best equipment online pharmacies. the company’s investments in enables us to This issue isn’t just facilities and new technologies produce tablets economic, it places are helping the pharma sector and capsules with an immeasurable reach its goals. milligrams of active number of lives product in the at risk. We’ve The View From the US safest of conditions. developed on- Increasingly, dose authentication What are the biggest challenges faced pharma companies technologies that by your customers? want to work with enable drug products CC: Speed of development is a major partners who have to be instantly verified with concern across the pharma industry. We’re continuous manufacturing track and trace across the entire operating in a climate where companies need capabilities because of the flexibility supply chain, down to the individual pill. to have ready access to reliable and trusted of the technology and other benefits. Patient compliance is an important area partners that can accelerate processes, but We’ve invested in continuous coating and for the industry to improve. With larger fixed still provide robust formulations. Though continuous powder feeding equipment or combination doses, some patients find it companies may want faster development, so we can stay abreast of the changing challenging to swallow their medication they also need to ensure that the best pharmaceutical manufacturing landscape. As due to size. Even single dosages can be formulations – with the most appropriate a result of our commitment, we can now go challenging for elderly and pediatric dosage forms – make it to market. from the API all the way to a finished coated populations. Colorcon has addressed As a result of the pandemic, on-site dosage form within our facilities and provide this through the development of easy- staff numbers have been lowered in many prototypes ready for customer evaluation. to-swallow coatings that improve the pharma companies – and so too has the patient experience, making it easier amount of time spent in the lab and research Why is your global support network to take medication and adhere to facilities. During this period, we’ve heard of so important? treatment regimens. companies taking longer to achieve their CC: Many of our customers work in The demand for clean label products project goals simply because of challenges multinational companies with locations continues to increase with consumers with resources. And that’s where we have across the world. With our network of wanting wholesome ingredients they can been able to help. In many cases, we have 22 technical service labs, we are close to understand, know, and trust. For many conducted formulation and coating trials for our customers – allowing us to provide consumers, the term “label friendly” our customers. Their product development tailored and consistent support, wherever means the elimination of a long chemical teams relied on us to provide resources they are. We recently conducted trials in our ingredient list and includes those that are through our labs when they didn’t have full California lab and had virtual attendance by familiar with consumers and are naturally access to their own. We’ve had our learning our North American team, as well as our derived minimally processed sustainable lifecycle; from formulation development best practices, helping our customers Meet the Experts through to scale-up and manufacture. with product selection, development and Alongside our teams, we make sure troubleshooting. Through working closely Charlie Cunningham, that our labs are top-notch and with the professionals in our laboratories, Technical Director North that our people have the latest where our samples are made and trials America, Colorcon digital and virtual technologies conducted, we ensure that our customers “In addition to the to conduct formulation receive best-in-class services and products products that we formulate, and coating trials with in a timely way. As part of a wider global manufacture, and sell, our customers.” team, we are all charged with ensuring that we also provide hands-on our innovative products, new specialist technical support to Jason Teckoe, Technical initiatives and applications our customers – whether that be Director EMEA, Colorcon data are implemented in their labs or within one of our facilities. “I manage and support around the region. I love Our network of technical managers our team of technical the variety that comes with provides expert assistance to support managers across the this role. No two days – or our customers through the entire product EMEA region – sharing customers – are the same!”
ingredients. With our most recent launch insights that we can provide to customers with Colorcon’s experts, the color, size, of Nutrafinish®, Label Friendly Coatings, during the early phases of their product and shape of tablets can all be selected Colorcon now provides best-in-class development, the better! to create market-leading products. Simply coatings to meet consumer needs for simple put, our services cover a broad spectrum ingredients and a clean label. How do you accelerate formulation of needs, ultimately helping speed up development? time-to-market, particularly for customers The EMEA Perspective JT: HyperStar t ® is a digital tool that who don’t have their own facilities. jumpstarts formulation development. The What do your clients need for complimentary service allows clients to What about the scale-up step development support? share details of their ideal dosage form, to manufacturing? JT: Alongside big pharma, there are API, and desired release profile with us, JT: . Essentially, scale-up comes down to good a growing number of virtual or small so that our technical team can determine process understanding and access to suitable companies developing drug products the best starting formulation. In essence, equipment. We have technical experts and that don’t necessarily have their own lab HyperStart references a broad spectrum equipment available across the EMEA region capabilities or resources to progress their of formulation data to help reduce to help customers in both the early and products into the clinic. These companies complexity and make the development the latter stages of product development often work with consultants and CROs to process as efficient as possible. and can support standard pharmaceutical help convert their drug substance into a processes such as compression, granulation, drug product suitable for clinical supply and How else are you helping customers and coating. This is also important for early trials. With our understanding of excipients reduce their timelines? phase development, when companies may and coatings, we can support early phase JT: The great wealth of knowledge and not have the right equipment or capacity screening activity, providing these virtual or experience in our team can help accelerate to progress trials. small companies access to our formulators, development in myriad ways. We provide equipment and services as well as generating regulatory insight and documentation Why is it so important for Colorcon to non-GMP prototypes of the dosage form to support smooth submissions for new continue to invest to support clients? for subsequent clinical supply. products and supplementary filings. JT: The pharma industry is constantly To expand our capability to meet Companies don’t want to encounter changing and evolving. We are investing customer needs, we’ve invested in flexible regulatory hurdles in any region that they to keep ahead of demand and help our isolators for our Technical Centre at our intend to market in, so we’re always on customers to deliver their business goals. Dartford, UK site, so that we have the ability hand to provide insight and guidance. Our investment in technology, new to handle more potent APIs, and added Our tablet design service, BEST®, also capabilities, and professional expertise small scale characterization equipment to helps ensure that our customers’ products means that we can help our customers evaluate flow, granulation, compression stand out from the competition and are through the challenges of today and in and encapsulation behaviour. The more designed with the patient in mind. Consulting the years ahead.
www.colorcon.com 34 NextGen
NextGen
R&D pipeline New technology When the Future trends Chips Are Down
Looking for human-relevant information on an early drug candidate? Organ-on-chip technology can offer new insights.
By Stephanie Sutton
Organ-on-a-chip is considered an Organ-on-a-chip technology can be living human epithelial cells on the important emerging technology for designed to fully recreate the complex, upper channel and living human organ- research and development because it dynamic state in which living cells specific endothelial cells on the lower can show how a candidate drug might function within a human organ – a channel. Such an arrangement recreates react in human tissue structures – as so-called “home away from home” for the essential tissue-to-tissue interface opposed to traditional animal models human cells. Such an environment found in every organ. The channels – long before it enters the clinic. Here, involves modelling complex interactions, are flanked by vacuum channels that we speak with Lorna Ewart, Executive often in three dimensions. This starts can provide tissue-relevant mechanical Vice President, Science of Emulate, to with the extracellular matrix (ECM), forces for organs to mimic processes such learn more. which is optimized for organ-relevant as breathing in the lung. composition, stiffness, and cell How do organ-on-chip platforms attachment. Homotypic and heterotypic How well used is organ-on-a-chip accurately mimic human organs? cell-cell interactions are recreated. And technology in drug discovery and the Organ-on-a chip technologies use finally, the model seeks to create oxygen, pharma industry today? microscale engineering technologies nutrient, and chemical gradients that We’re working with around 19 of the top combined with cultured living human provide the complete set of cues cells 25 pharmaceutical companies as defined cells to create an experimental platform need to behave and function accurately by their R&D spend. In my view, the that recapitulates the physiological and – just as they would in the human body. industry should always be considering mechanical microenvironment of whole As an example, Emulate Organ-Chips the adoption of new technologies living organs. These microphysiological are composed of a transparent, flexible that can reduce the long, costly drug systems (MPS) enable the study silicone polymer (polydimethylsiloxane discovery and development process, or of complex human physiology and (PDMS)) about the size of an AA improve confidence in how a medicine pathology in an organ-specific context. battery. Each chip has two parallel, will perform in human trials. These specialized in vitro human disease fluidically independent channels Organ-on-a-chip technology can models can revolutionize drug discovery separated by a flexible membrane, with develop human-relevant data for drug and development by helping researchers pores seven microns in diameter. The candidates and also offer a sustainable, better predict human responses to membrane is coated with an optimized ethical research model by relying less on medicines, including safety and efficacy. ECM before the channels are lined by animal models – which are frequently NextGen 35
shown to poorly recreate the human of the drug discovery and development standardized, enabling broader inter- pathophysiological phenotype. process. One notable example is the laboratory comparisons, technology Organ-on-a-chip technology is barely human Blood Vessel-Chip developed by adoption for key applications, and more a decade old, so the pharma industry is Emulate in collaboration with Janssen widespread use. still in the testing and evaluation phase. Pharmaceuticals, which was able to Second, regulatory acceptance Around 23 pharmaceutical companies recreate the thrombotic toxicities of a will trigger technology adoption, have established an MPS affiliate under monoclonal antibody drug that led to especially relating to robustness and the IQ Consortium – which aims to its failure in human clinical trials (2). reproducibility (both inter- and intra- advance innovation and quality in the The ultimate goal of some pharma laboratory) of the technology. This is a biopharmaceutical industry. The goal of companies is to include organ-on-a-chip central component of the Cooperative this MPS affiliate is to define the criteria data within their regulatory filings for Research and Development Agreement of use for new, sophisticated in vitro new drugs, to augment the results from (CRADA) between Emulate and the models, such as organ-on-chip and, to this animal data or to strengthen a weight- FDA. For example, if organ-on-a-chip end, a series of organotypic manuscripts of-evidence argument. technology is proposed to replace an have been published to guide technology existing approach, regulators will want innovators, such as ourselves, toward What is necessary to promote to see concordance of data between building organ-models to address the greater uptake? the organ-on-a-chip model and the key safety- or ADME-related questions Promoting greater adoption of organ- animal model that it intends to replace. relevant in drug development (1). The on-a-chip technology is likely to result In other cases, if the use is related to affiliate is also closely aligned with the from progress in several areas. These a greater understanding of mechanism FDA, thereby ensuring consistency of have been discussed in greater detail by of action, the user needs to articulate direction and approach for advancing in Nature Reviews Drug Discovery (3). which regulatory question is being this new technology. First, adoption will be driven by a move answered. A good example of this could In my experience, customers are toward technology standardization. be explaining species differences with really interested in using organ-on-a- Because this is still a developing field, respect to toxicological outcome, as has chip technology for internal decision- there are multiple MPS platforms and been nicely described in a paper where making purposes within the preclinical approaches. As leading technologies are the Emulate Liver-Chip was used to safety testing or target validation stages further industrialized, they will become understand drug toxicity (4).
www.themedicinemaker.com 36 NextGen
the existing regulatory framework for product testing. The FDA seeks to engage with alternative methods not only to reduce the risk of products reaching the market with toxicological consequences, but also to ensure that regulated products reach the market faster and with improved efficacy (6). The first FDA-Emulate CRADA was in 2017 and was focused on toxicology. Our new multi-year CRADA expands beyond toxicology and is agency-wide, meaning that divisions throughout the FDA will be able to use Emulate products for programs that address some of the most challenging areas, including Alzheimer’s disease, the intestinal microbiome, liver toxicity, and, importantly, COVID-19 (5). Third, significant progress is Our new multi-year CRADA expands Toxicology leaders at the FDA have underway in demonstrating confidence beyond toxicology and is agency-wide, been involved in collaborative research in new organ-on-a-chip models, as meaning that divisions throughout with our technology since before we well as translational qualification. the FDA will be able to use Emulate commercialized it – back in 2010 when it Importantly, the work of the IQ MPS products for programs that address was in development at the Wyss Institute affiliate is designed to articulate new some of the most challenging areas, for Biologically Inspired Engineering at roadmaps that are sound and relevant including Alzheimer’s disease, the Harvard University. In 2017, after years for enabling organ-on-a-chip innovators intestinal microbiome, liver toxicity, of being connected with our technology, to navigate towards faster development and, importantly, COVID-19 (5). the FDA established an initial CRADA and broader adoption. Essential criteria Toxicology leaders at the FDA have focused on using Emulate Organ-Chips to be fulfilled include the ability to been involved in collaborative research for toxicology applications within the reproduce functionality within and with our technology since before we regulatory process. It was designed so the across laboratories, execute with good commercialized it – back in 2010 when it FDA could gain first-hand experience experimental design, knowledge of was in development at the Wyss Institute with our Human Emulation System, model stability over time and donor for Biologically Inspired Engineering at working alongside our scientists to variability, and qualification of Harvard University. In 2017, after years generate data together. It is beneficial for supporting analytical methods. of being connected with our technology, the FDA to have a Human Emulation Lastly, successful proliferation of the FDA established an initial CRADA System in their laboratories, and it will organ-on-a-chip technology – and the focused on using Emulate Organ-Chips pave the way for qualifying the use of experimental results they produce – for toxicology applications within the our technology and integrating it within within pharmaceutical organizational regulatory process. It was designed so the the existing regulatory framework for hierarchies and cultures will lead FDA could gain first-hand experience product testing. to the continuing growth trajectory with our Human Emulation System, The FDA seeks to engage with of adoption. working alongside our scientists to alternative methods not only to reduce generate data together. It is beneficial for the risk of products reaching the market What can you tell us about the new the FDA to have a Human Emulation with toxicological consequences, but also CRADA with the FDA? System in their laboratories, and it will to ensure that regulated products reach The first FDA-Emulate CRADA was pave the way for qualifying the use of the market faster and with improved in 2017 and was focused on toxicology. our technology and integrating it within efficacy (6). NextGen 37
The first FDA-Emulate CRADA was differences in viral pathogenesis, www.iqmps.org/publications in 2017 and was focused on toxicology. immune response and immune system 2. R Barrile et al., “Organ-on-Chip Recapitulates Our new multi-year CRADA expands interactions, inflammatory response, Thrombosis Induced by an anti-CD154 beyond toxicology and is agency-wide, pharmacokinetics, and drug action – are Monoclonal Antibody: Translational Potential of meaning that divisions throughout known to impact successful translation Advanced Microengineered Systems,” Clin. the FDA will be able to use Emulate of preclinical data to the clinic. More Pharmacol. Ther., 104 (2018). DOI: 10.1002/ products for programs that address human-relevant alternatives, such as cpt.1054 some of the most challenging areas, organ-on-a-chip technology can be 3. L Ewart, A Roth, “Opportunities and challenges including Alzheimer’s disease, the adopted to accelerate discovery and with microphysiological systems: a pharma intestinal microbiome, liver toxicity, development efforts in COVID-19 (7). end-user perspective,” Nature Reviews Drug and, importantly, COVID-19 (5). Historically, animal alternatives for Discovery (2020). DOI: 10.1038/d41573-020- Toxicology leaders at the FDA have studying respiratory viruses have involved 00030-2 been involved in collaborative research conventional in vitro models that use cell 4. K-J Jang et al., “Reproducing human and with our technology since before we lines (such as Vero, A549, or MDCK cells.) cross-species drug toxicities using a Liver-Chip,” commercialized it – back in 2010 when it These approaches are poor predictors of Sci. Trans. Med., 11 (2019). DOI: 10.1126/ was in development at the Wyss Institute human outcomes, most likely due to the scitranslmed.aax5516 for Biologically Inspired Engineering at simple culture conditions that lack the in 5. Emulate, “Emulate Signs Collaborative Harvard University. In 2017, after years vivo relevance and biological complexity Agreement with the FDA to Apply Lung-Chip to of being connected with our technology, required to model viral pathogenesis. Evaluate Safety of COVID-19 Vaccines and the FDA established an initial CRADA In contrast, Emulate Lung-Chips can Protective Immunity Against SARS-CoV-2,” focused on using Emulate Organ-Chips more faithfully recreate human biology (2020). Available at https://bit.ly/3srvr8j for toxicology applications within the and pathophysiology observed in vivo, 6. FDA, “Advancing Alternative Methods at FDA,” regulatory process. It was designed so the including mucociliary clearance, lung (2021). Available at: https://bit.ly/3de1xQa FDA could gain first-hand experience inflammation, immune cell recruitment, 7. Emulate, “How Organs-on-Chips Technology is with our Human Emulation System, cytokine production, viral infection, and being used to Provide Scientific Insights into working alongside our scientists to pulmonary edema (4, 8, 9, 10, 11, 12). COVID-19,” (2020). Available at generate data together. It is beneficial for Emulate Lung-Chips have been https://bit.ly/31i7Mve the FDA to have a Human Emulation shown to support key hallmarks of 8. D Huh et al., “Reconstituting organ-level lung System in their laboratories, and it will the cytopathology and inflammatory functions on a chip,” Science, 328 (2010). DOI: pave the way for qualifying the use of responses observed in human airways after 10.1126/science.1188302 our technology and integrating it within viral infection with human rhinovirus, 9. D Huh et al., “A human disease model of drug the existing regulatory framework for influenza, and SARS-CoV-2 viral toxicity-induced pulmonary edema in a product testing. particles that express the SARS-CoV-2 lung-on-a-chip microdevice,” Sci. Transl. Med., 4, The FDA seeks to engage with spike protein, as well as relevant levels of (2012). DOI: 10.1126/scitranslmed.3004249 alternative methods not only to reduce angiotensin converting enzyme-2 (ACE- 10. M Kasandra et al., “Duodenum Intestine-Chip the risk of products reaching the market 2) and TMPRSS2 protease. Emerging for preclinical drug assessment in a human relevant with toxicological consequences, but also evidence also suggests that the endothelial model,” eLife (2020). DOI:10.7554/eLife.50135 to ensure that regulated products reach tissue plays a critical role in SARS-CoV-2 11. J. Nawroth et al., “A Micro-Engineered Airway the market faster and with improved pathogenesis (13) – and some organ-on- Lung-Chip Models Key Features of Viral-Induced efficacy (6). a-chip products can incorporate this cell Exacerbation of Asthma,” [preprint] (2020). DOI: layer. Overall, Emulate Organ-Chips can 10.1101/2020.02.02.931055 In what ways can organ-on-a-chip enable more rapid insights into COVID-19 12. L Si et al., “Human organs-on-chips as tools for technology be used in the fight against and other human diseases – and precisely repurposing approved drugs as potential influenza COVID-19? predict human response to vaccines and and COVID19 therapeutics in viral pandemics,” The COVID-19 pandemic has required drug candidates. [preprint] (2020). DOI: scientists to improve the predictive 10.1101/2020.04.13.039917 nature of preclinical models to accelerate References 13. Z Varga et al., “Endothelial cell infection and therapeutics and vaccines entering the 1. IQ Microphysiological Systems Affiliate, endotheliitis in COVID-19,” The Lancet, 395 clinic. Species differences – including “Publications” (2020). Available at (2020). DOI: 10.1016/S0140-6736(20)30937-5
www.themedicinemaker.com 38 NextGen
Fifty Functional Cures Subscribe for free Gene therapy’s “single most remarkable achievement,” at: www.texer- CARs for solid tumors and plenty of new collaborations… enewsletters.com/ What’s new from the Cell + Gene Curator? cellandgene
By James Strachan
There have been some big moments In May, scientific attention returned these trials and he is highly deserving of for the gene therapy field – China's to the ADA-SCID field with what this award." approval of Gendicine in 2003, Fyodor Urnov, Professor and CRISPR And from clinical successes to, uniQure's European approval in 2012, researcher at the University of hopefully, a glimpse into the future… and Lexturna in 2018... But it would be California, described as “The single most a gene therapy has prevented learning difficult to top the moment that kicked remarkable achievement of the gene and memory loss in a mouse model of everything off: at 12:52pm, September therapy field since it started in 1989” (2). Alzheimer's disease (AD), according to 14, 1990, Ashanti DeSilva became the Donald B. Kohn and colleagues treated research from the University of California first person in the world to receive a 50 patients with ADA-SCID with an San Diego School of Medicine (5). The gene therapy (1). DeSilva was then investigational gene therapy composed of researchers used a viral vector to introduce four years old and had been diagnosed autologous CD34+ HSPCs transduced synapsin-Caveolin-1 cDNA into the with adenosine deaminase (ADA) ex vivo with a lentiviral vector encoding hippocampus region of three-month- deficiency, a form of severe combined human ADA (3). Overall survival was old transgenic AD mice. Caveolin-1 immunodeficiency disease (SCID). 100 percent up to 24 and 36 months. is a scaffolding protein that builds cell Over the first six months following And of the 50 children, 48 are no longer membranes; when it decays, it causes cell treatment, DeSilva T-cell count showing symptoms of ADA-SCID. dysfunction and neurodegeneration. increased and she quickly tested at Bravo to everyone involved in the work! “These results suggest SynCav1 gene normal levels. Her health took a And special congratulations to Kohn, who therapy is an attractive approach to remarkable uptick over the following two this week was awarded the ISCT Career restore brain plasticity and improve brain years. But with no selective advantage, Achievement Award in Cell & Gene function in AD and potentially in other the small number of transfected T cells Therapy (4) – the Society’s highest honor. forms of neurodegeneration caused by were unable to form a stable population “He developed the first successful unknown etiology,” wrote the authors (6). and eventually died. However, DeSilva hematopoietic stem cell gene therapy, exhibited no noteworthy side effects and for children with 'bubble baby disease' Cracking solid tumors grew up normally into adulthood. The – ADA deficiency – and has now Many people have spoken about the trial showed that gene therapy could be functionally cured over 50 children,” shift, following the first CAR-T safe and effective – though, in this case, said Jan Nolta, UC Davis. "The entire approvals in 2017, from questions of not curative. field seeks to build upon his success in scientific efficacy to manufacturing and NextGen 39
Credit: Matt Beardsley/UCSF (PRNewsfoto/ Thermo Fisher Scientific)
So, what exactly is How do you keep Meet The Curator The Cell + Gene up with all the Curator? developments How did the Cell + Gene Curator The idea behind in cell and gene come about? The Curator therapy? I joined Texere Publishing and The is to deliver It’s easier said Medicine Maker magazine around five the week’s cell than done! I years ago. Back then, we were thinking and gene therapy have a long list of in terms of small molecules and news – the latest sources that I check biologicals, which included cell and gene discoveries, process throughout the week therapies. Now, given the rise of CGT, innovations, and – filing away anything we see the pharma industry as a triad: business deals – into a that sounds interesting small molecules, large molecules, and five-minute read. It’s aimed before whittling the stories down advanced therapies. We realized when at professionals working in the cell to the top 15 or so that make it into The looking for content for our Upfront and gene therapy sector. But we also Curator. But I find the easiest way to keep news section that there were more want the Curator to be a hub for the up with all the developments is to speak CGT-related stories – be it new facilities community – somewhere to share ideas with people. I spend a lot of time reaching being built, clinical updates, early-stage and opinions in addition to curating all out to my network in the field to make sure research or new collaborations – than the business and scientific updates. So I’ve got my ear to the ground. And we’re we knew what to do with. I remember I’m always keen to shine a light on the frequently developing longer-form CGT- someone asking the question: wouldn’t big issues facing the field, as well as related pieces for The Medicine Maker, it be useful if someone just collated – or reaching out to the individuals behind which are usually based on interviews – curated – all the most interesting stories the news of a new manufacturing facility and that’s an excellent way to keep up with each week? This was the genesis of The or new class of CAR T-cell therapy. what the experts and pioneers in the field Cell + Gene Curator. are thinking (or worried!) about.
commercialization – how do you scale a participant in a phase I clinical study such as ovarian and lung (11). personalized, autologous, cell therapy? assessing the safety of CAR macrophages The new approach has two-steps. The Will the industry shift to a more (8). A review of recent developments in first step uses synNotch to give CAR-Ts decentralized manufacturing model? CAR-macrophage-based treatments the ability to carefully “judge” if they are And how will payers afford the often for solid tumors from Anhui Medical in a tumor or not; the second step uses very expensive one-off costs? Several University, China, researchers cited a different set of synNotch sensors to years later, many of these questions “great potential” – with issues around ensure a strong tumor-killing response. remain unanswered. But it seems we’re cell proliferation and migration (9). “These findings address all critical witnessing a return to questions of In April, the University of California challenges that have been in the way scientific efficacy – specifically, how can San Francisco published two papers of developing immunotherapies for we crack solid tumors? on their “SynNotch” system. In the patients who suffer from these cancers,” Last year, University of Pennsylvania first paper, they found that SynNotch- said Hideho Okada, co-author of the researchers genetically engineered CAR-T cells could completely clear first paper. “This science is ready to move macrophages to kill solid tumors in both human patient-derived tumors from towards clinical trials.” mouse models and human samples (7). the brains of mice – safely and without Then, in March, Carisma Therapeutics, recurrence (10). In a second paper, Collaboration is key a company founded by researchers at the another set of researchers showed A cross-industry alliance between Astellas, University of Pennsylvania, announced how components of the system can be bluebird bio, Bristol Myers Squibb, Kite that it had dosed its first human switched out to target other cancers, (a Gilead company) and Novartis called
www.themedicinemaker.com 40 NextGen
the Cell and Gene Collective launched in scale up development and accelerate therapy preserves neuronal and synaptic May. Their (collective) aim is to increase commercialization and manufacturing. morphology and prevents neurodegeneration in awareness and understanding of cell and Ultimately, this new facility will help a mouse model of AD,” Mol Th, 21, 434-450 gene therapies in the UK. Earlier this year, more cell therapies progress through (2021). PMID: 33981778 we spoke with Roudie Shafie, Director the pipeline to deliver transformative 6. Eurekalert, “Gene therapy in alzheimer's Access Our at OVID Health (which is acting as medicines to patients.” disease mouse model preserves learning and secretariat and coordinating the efforts R&D Eye Alliance. BlueRock, memory” (2021). Available at: Capabilities to of the Collective) about the project (12). FUJIFILM, and Opsis also entered https://bit.ly/3wzSijT And we spoke with Anita Ralli, Chair into a strategic R&D alliance in May 7. M Klichinsky et al., “Human chimeric antigen of the Collective and Associate Director to discover cell therapies for eye diseases receptor macrophages for cancer Reduce Your Government Affairs at Gilead Sciences, to (14). The companies have their sights set immunotherapy,” Nat Biot, 38, 947-953 find out why collaboration is so important on off-the-shelf iPS cell therapies for (2020). DOI: 10.1038/s41587-020-0462-y for cell and gene therapy. age-related macular degeneration and 8. PR Newswire, “CARISMA Therapeutics to Development Time “To date, the UK has been pioneering inherited retinal diseases. Under this Present Data at The American Association for in adopting cell and gene therapies agreement, BlueRock has the option Cancer Research Annual Meeting” (2021). to transform the lives of people with to exclusively license three retinal Available at: https://prn.to/3bTkh68 Getting Started – devastating diseases, such as end stage cell therapies currently in pre-clinical 9. Y Chen et al., “CAR-macrophage: A new Proof of Concept for Oral Solid Dosage Forms cancer,” she said. “However, as the development from both parties. immunotherapy candidate against solid science develops and these technologies Finally, we return to solid tumors: MD tumors” (2021). Available at: Access confi dential and complimentary concept are applied to a greater number of Anderson and Refuge Biotechnologies https://bit.ly/2RRto0r formulation design, supported with characterization conditions, new models of care will need are working together on engineered 10. JH Choe et al., “SynNotch-CAR T cells capabilities and analytical services, directly with Colorcon experts. to be developed and potentially new ways TILs and CAR T-cell therapies for solid overcome challenges of specificity, heterogeneity, of accessing value established. Through tumors (15). MD Anderson will apply and persistence in treating glioblastoma,” Sci sharing our experience across companies, Refuge’s cell engineering platform to its Trans Med, 13, 591 (2021). DOI: 10.1126/ Best Fit Excipients – we hope to be constructive partners to the TIL programs, and the companies will scitranslmed.abe7378 Process and Characterization Capabilities NHS and other stakeholders in helping co-develop Refuge’s RB-340, a HER-2 11. A Hyrenius-Wittsten et al., “SynNotch CAR Colorcon provides extensive experience and technical knowledge for ensure people in the UK continue to be targeted CAR T. circuits enhance solid tumor recognition and the formulation development of oral solid dosage forms—immediate, among the first to access the innovation “It is my belief that TILs are poised for promote persistent antitumor activity in mouse modifi ed and targeted release. We provide proof of concept formulation these therapies represent.” a significant impact in the field of cancer models,” Sci Trans Med, 13, 591 (2021). DOI: development, and then in turn assist clients with scale-up development, In further collaboration news, Thermo therapy, and engineering improved TILs 10.1126/scitranslmed.abd8836 process optimization and technology transfer. Fisher Scientific and the University of is a vital part of advancing this modality,” 12. The Medicine Maker, “Spreading the Cell and California, San Francisco are working said Jason Bock, Vice President of Gene Word” (2020). Available at: Our technical expertise supports a robust and scalable manufacturing together on a cell therapy manufacturing Therapeutics Discovery and Head of https://bit.ly/3hX6I9O process, plus regulatory support with fast online access to and collaboration center. Set to open in Biologics Development at MD Anderson. 13. PR Newswire, “Thermo Fisher Scientific and documentation to minimize fi ling delays. 2022, the 44,000-square-foot facility University of California, San Francisco to will be located on UCSF's Mission Bay References Open Cell Therapy cGMP Manufacturing and campus (13). We reached out to Mark 1. GEN, “Making History with the 1990 Gene Collaboration Center” (2020). Available at: Reach Your New Drug Product Milestone Faster with Colorcon Stevenson, Executive Vice President Therapy Trial” (2016). Available at: https://prn.to/2SylxF6 and Chief Operating Officer of Thermo https://bit.ly/2Tiphee 14. Bluerock, “BlueRock Therapeutics, FUJIFILM · Access to Experts and Equipment for Trials Fisher Scientific, to find out more. 2. Twitter (2021). Available at: Cellular Dynamics, and Opsis Therapeutics · Processing and Characterization Capabilities “Emerging biotech companies are https://bit.ly/3hOUMqu Enter into a Strategic R&D Alliance to doing a great deal of innovation in the 3. DB Kohn et al., “Autologous Ex Vivo Discover and Develop Cell Therapies for Eye cell-based therapies space, but they Lentiviral Gene Therapy for Adenosine Diseases” (2021). Available at: often face challenging timelines and Deaminase Deficiency,” NEJM (2021). https://bit.ly/3uo8HGr milestones to support their programs,” PMID: 33974366 15. MD Anderson, “MD Anderson and Refuge he said. “Cell-based therapy developers 4. ISCT, “Congratulations to the 2021 ISCT Biotechnologies collaborate to advance From Core to Coating, will also have the opportunity to integrate Award Winners” (2021). Available at: next-generation cell therapies for treatment of high-quality materials, instruments and https://bit.ly/3bXisoW solid tumors” (2021). Available at: Your Formulation Partner of Choice pre-configured workflows designed to 5. S Wang et al., “Synapsin-caveolin-1 gene https://bit.ly/3bWLFQQ www.colorcon.com
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12198 Colorcon Ad May 2021_v8.indd 1 01/06/2021 11:07 42 Business Business
Economic drivers Emerging trends How to Plug Business strategies the Skills Gap
Experts from the BIA got together to discuss how the UK’s biotechnology industry can capitalize on recent investment and post-COVID-19 public appreciation to attract and train more people
By James Strachan
The UK’s biotech sector is booming. “That’s a crazy number,” he said. But trained people in process development, According to the Bioindustry Association it’s not just science and manufacturing manufacturing, data handling, and (BIA)’s recent report of UK biotech skills; there’s also a need for managers, software architecture, as well as specialists financing, there has been a 1,000 percent leaders, software developers, commercial in commercialization. She also noted that increase in biotech investments since 2012 and financial skills, regulatory, personal cross-movement of staff from company (1) – and the UK is up there with San development, and more. “We really need to company is leading to continuity gaps Francisco and Boston as one of the world’s to think about how to fill these gaps.” and rising salaries. leading life science clusters. The sector has The panel also discussed the challenges “This makes the UK less attractive to also been seen as a shining light in the fight facing specific sectors within the life investors wanting to set up new cell and gene against COVID-19, with several UK-based sciences, beginning with cell and gene therapy companies,” she said. Delahaye also companies and institutions playing key roles therapy. The UK Cell and Gene Therapy spoke about the challenges universities have in developing and manufacturing vaccines Catapult’s Skills Demand Survey 2019 faced during the pandemic and how those quickly. However, despite the positivity and found that 83 percent of ATMP and could exacerbate the problem. “Universities momentum within the sector, there is a lack vaccine manufacturing companies have not been able to offer much lab time of skills in certain areas – threatening the raised concerns that the recruitment and for students going through undergraduate industry’s potential growth. retention of skilled talent would slow down courses during the pandemic and that could or delay their forecasted manufacturing add to the skills gap,” she said. “It's a bit of The scale of the problem expansions (3). And the problem isn’t a bleak picture.” To characterize the scale of the skills limited to the UK. For example, the The panel identified genomics as challenge and offer suggestions for International Society for Cell and Gene another field with positions to fill. plugging the gap, the BIA hosted a panel Therapy (ISCT) has put together a range Adrian Ibrahim, Chair of the BIA discussion entitled, “How to develop of proposals to help address the global Genomics Advisory Committee and the skills needed for a thriving biotech talent gap in advanced medicine, including Head of Technology Transfer and sector.” mentoring programs, training courses, and Business Development at the Wellcome Will Milligan, Chair of the BIA’s LeaP public education programs, which The Sanger Institute, believes genomic data Alumni Group and Process Development Medicine Maker covered in its Cell and is only going to get bigger and more Lead at eXmoor Pharma Concepts, set Gene Therapy Supplement (4). complex. “As that data grows, we need the scene by citing the BIA and the Reporting on findings from the smarter ways of aggregating, analyzing ABPI’s Life Sciences 2030 Skills Strategy BIA Cell and Gene Therapy Advisory or visualizing [it],” he said. “There is a report (2). Their research found that the Committee (which she chairs), Helen massive and already unmet need for UK will need 133,000 skilled scientists Delahaye, Consulting Chief Operating high-quality data scientists and high- by 2030 to support the industry’s growth. Officer at Azellon, highlighted a lack of quality software engineers. Not wishing to sound negative, but we talked about importance of apprenticeship schemes Training Centres, which will deliver this stuff five, six, seven, eight years ago – of which there are several in the UK. on-site advanced therapies and vaccine – it wasn’t difficult to predict. We have Responding to the recommendations of manufacturing training – both practical some initiatives that we put in place, but the Advanced Therapies Manufacturing and digital. In March, the National not enough to keep pace with genomics.” Taskforce, the UK Cell and Gene Therapy Horizons Centre, Darlington, was The third sector discussed by the Catapult was awarded £1.5 million by the confirmed as the first National Training panel was engineering biology – the UK government’s Industrial Strategy Centre (5); and in May, RoslinCT, design and construction of novel genes, Challenge Fund to establish the Advanced Edinburgh, was selected as the second (6). pathways, and organisms. It brings Therapies Apprenticeship Community Delahaye also discussed a number of together a range of disciplines and skills (ATAC). The purpose of the community training videos and webinars that have from biology to chemistry, engineering is to create a ready supply of skilled talent been created in partnership with the to bioinformatics and computing – ranging from manufacturing operatives NHS. “These have become very popular, and that means competing with other, to technical experts and researchers. Each attracting up to 1,800 attendees,” she said. often lucrative, sectors for prospective Friday, ATAC hosts a “lunch-and-learn” The group also hosts a series of e-learning employees. Tim Brears, CEO of meeting for employers who already have modules, which are free to NHS staff and Evonetix, used his company as an apprentices or who are thinking of taking can be part of their individual professional example. “We’re developing a platform apprentices on. “That’s one great initiative development as certified courses. for DNA synthesis and most of the that’s helping to bridge the skills gap in For students looking to get into the people within our company are physical cell and gene therapy,” said Delahaye, industry, there are now postgraduate scientists; we have electronics engineers, who also noted that there are currently courses in cell and gene therapy in the UK software engineers, chemists, biologists – around 137 apprentices working in six – notably at the University of Manchester, a wide range of disciplines,” he said. “If companies, half of which are SMEs. “But the University of Sheffield, and University you contrast that to the traditional drug that’s still a small number compared with College London. And, in the US, discovery company, where you might the 33,000 scientists needed.” Delahaye noted that some companies find medicinal chemists, biologists, and Another piece to the puzzle is the have collaborated with community so forth, the skill sets we need are much Advanced Therapies Skills Training colleges to set up vocational degrees that broader. We’re competing with the likes Network (ATSTN), which is backed train technicians in manufacturing and of Google and Microsoft.” by £4.7 million in funding from the process development for cell and gene Department for Business, Energy & therapies. “This is something that could How can we close the gap? Industrial Strategy and Innovate UK. be considered in the UK,” she said. Coming back to cell and gene therapy, Over the past year, ATSTN announced Reaching further back, Delahaye noted Helen Delahaye spoke about the two of an initial three new National that a “cell and gene therapy explainer”
www.themedicinemaker.com 44 Business
has been well received in schools. “It was from their sites. It gives participants scale, cross-disciplinary work.” actually written for politicians,” she said. a chance to network with others and We also reached out to Oliver “We need to be thinking about our school draw upon different experiences and Hardwick from Cytiva, and Chair of leavers at 16 and 18. Where are they backgrounds. “How often do people from the BIA Skills Network, who summed headed? Are they aware of the industry?” supply chain manufacturing or regulatory things up by painting a positive picture When asked what could be done to help backgrounds get together and talk across for the future of the sector. “This past Reliable scale-up academia and industry build their skills in sectors and across companies?” he asked. year, the UK Life Sciences sector has the genomics sector, Adrian Ibrahim cited Milligan previously participated in the demonstrated its capabilities to the world. the need for pre-competitive knowledge program while working for a 30-person And with appropriate commitment from from lab-scale sharing. The Wellcome Genome Campus company and had the chance to visit sites government, we are likely to see rapid set up an initiative six years ago to like GSK, Allergan, and Fujifilm. growth in the biotech sector over the identify targets based on strong genetic “It really opened my eyes to what else next 10 years – with the UK positioned associations underlying disease. Industry goes on in the industry,” he said. “There alongside the world’s leading biotech to production partners and academics are involved – were some really inspirational moments regions,” he says. and everyone involved has a veto on the during the program. For example, we “At the core of that growth will specific programs. “Companies support the were visiting Fujifilm and Steve Bagshaw, be the skills and talent development program because they can influence which who was CEO at the time, walked in and across all aspects of our industry – from SB10-X programs are taken while also accessing chatted to us for 30 minutes about his career bioinformatics and cGMP operations, genomics infrastructure – of which there and what he’d done in the industry. And it to entrepreneurial and business may be one or two in the world at that scale, was a fascinating story and those kinds of management skills. Continued focus on and certainly only one in Europe,” he said. moments kind of stick with you and inspire skills is required to ensure we build on “This has exposed hundreds of scientists to you going forward.” The program was so a position of strength and capitalise on quite a large number of pharma companies. successful that the BIA doubled its size after investment to propel the UK life science There are some of the finest minds in the two years – there are two parallel cohorts sector forward to the overall benefit of world working with each other, sharing now. “The key thing it does is inspire.” patients globally.” knowledge and building.” The discussion closed with the panelists Scale-up Ibrahim remembered speaking to the sharing their final thoughts on how to References while maintaining the quality head of cancer programs when he first close the skills gap within UK biotech. 1. BIA, “The science of success: UK biotech arrived at the Welcome Institute in 2012. “When we’re asking the question, we’re financing in 2020” (2021). Available at: The consistent hydrodynamics driven by Kuhner shaking https://bit.ly/3fGLSsy. “He said, ‘Some of our biologists will thinking 10 years from now, but my machines allow for an efficient scale-up from microtiter arrive without coding skills, but nobody feeling is that we need to start looking 2. Science Industry Partnership, “SIP 2030 Skills will leave without coding skills,’ and earlier,” said Charlotte Casebourne, Vice- Strategy” (2020). Available at: plates to shake flasks and production scales. Our orbitally- that’s how it should be across the board. Chair of the BIA People, Skills and Talent https://bit.ly/3bLYFZv. shaken bioreactors (OSB) for single-use bags expand the We have lots and lots of smart people; Working Group and Co-Founder & CEO 3. CGT Catapult, “UK wide sites announced as ISF1-Z Peltier range of shaken cultivations up to 2500 L. we just need to create the opportunities.” of Theolytics, echoing Delahaye’s earlier preferred bidders for National Training Centres point. “We need to be asking: at what for the Advanced Therapies Skills Training You will find further information at: LeaPing forward stage can the BIA start to inspire people Network” (2020). Available at: Adolf Kühner AG www.kuhner.com/en/kuhner-technology/scale-up.php Will Milligan spoke about the LeaP to start thinking about what their path to https://bit.ly/2T7tiCa. +41 61 319 93 93 Alumni Group as an example of a way to the industry might look like?” 4. James Strachan, “Next Level Cell and Gene www.kuhner.com inspire people to advance in their careers Delahaye noted that there are many Therapy,” The Medicine Maker (2019). and, potentially, become leaders. The British people working abroad in the Available at: https://bit.ly/3hPc1Il. program brings together 12 people per cell and gene therapy area. “How can we 5. ATSTN, “National Horizons Centre expands cohort from different companies within bring them back to the UK to decrease as National Training Centre for the Advanced the biotech sector. “It’s an incredibly our skills gap?” she asked. “Perhaps that’s Therapies Skills Training Network (ATSTN)” achieve your goals with Kuhner shaker valuable and unique opportunity,” said something we can take forward.” (2021). Available at: https://bit.ly/34Kpo4v Milligan. The program lasts two years and, “We have lots of small wins, but big wins 6. ATSTN, “RoslinCT has been selected as the every couple of months, each member of only come from national-scale programs,” second National Training Centre in the the program takes turns inviting the others said Ibrahim. “We need to start funding Advanced Therapies Skills Training Network” into their company to share best practice academies and specialist centers for large- (2021). Available at: https://bit.ly/3ihf4t1 Reliable scale-up from lab-scale to production
SB10-X
Scale-up while maintaining the quality
The consistent hydrodynamics driven by Kuhner shaking machines allow for an efficient scale-up from microtiter plates to shake flasks and production scales. Our orbitally- shaken bioreactors (OSB) for single-use bags expand the ISF1-Z Peltier range of shaken cultivations up to 2500 L.
You will find further information at: Adolf Kühner AG www.kuhner.com/en/kuhner-technology/scale-up.php +41 61 319 93 93 www.kuhner.com
achieve your goals with Kuhner shaker 46 Business
Powering on Through the Pandemic
We ask members of our Power List to share their views on how the industry is coping with the pandemic – and what needs to change for the future
We recently celebrated the publication of The COVID-19 while maintaining supply of for many candidates, but we continue to see Medicine Maker 2021 Power List, which their clients’ non-COVID-19 products, but huge promise in the development of cell and presented the top 20 most inspirational it remains to be seen how manufacturing gene therapies. From a CDMO perspective, medicine makers in three different categories infrastructure will change to accommodate that’s reflected in the billions of dollars – Small Molecules, Biopharmaceuticals, the potential for more global vaccination our members have invested in that space. and Advanced Medicine – as nominated programs. This will mean hard decisions These can be transformational modalities by readers and selected by a judging about building – and paying for – excess for patients, and given the expertise and panel. The full list is available at https:// capacity into the supply chain, even before capital necessary to manufacture them, themedicinemaker.com/power-list/2021. the drug substance stage. Government it’s clear that CDMOs will be critical in Here, we ask a selection of Power Listers interventions to reallocate resources to bringing that vision into reality. for their thoughts on the COVID-19 particular manufactures may help boost pandemic… production in the short term, but can create cascading disruptions of other critical, life-saving products. Avoiding those unintended consequences, stemming national or regional blockades, and ensuring equitable access to vaccines: those may be “beyond our pay grade” as CDMOs, but the pandemic has raised the awareness of the outsourcing sector and we’ve made sure Kiran Mazumdar-Shaw our voice is heard in these conversations. Kiran Mazumdar-Shaw is Executive Gil Roth Beyond that, the pandemic has created Chairperson at Biocon and Biocon Gil Roth founded the Pharma & changes to every aspect of our lives and Biologics. She is seen as a pioneering biotech Biopharma Outsourcing Association in work and it remains to be seen what entrepreneur, a healthcare visionary, a 2014 to give a voice to the CDMO sector in changes will prove long-lasting. The FDA, global influencer, and a philanthropist. regulatory, legislative, and business matters. for example, has had to make major changes During the pandemic, he helped connect in its inspection practices when in-person A positive outcome of the pandemic is the the federal government and other parties inspections were off the board. We’ve global commitment to collective surveillance with CDMOs to develop and manufacture had conversations with them and other and pandemic prevention and preparedness. treatments and vaccines. regulators about how to incorporate some At long last, healthcare investment is likely of their changes – more records requests, to see a sharp increase across the world. Post-pandemic, the industry – and use of virtual technologies, greater reliance Moreover, the crisis has brought to the governments across the world – will have on mutual recognition of inspections – into fore a host of complex issues around drug to answer major questions about pharma the post-pandemic world. Greater flexibility pricing and distribution, as well as a number supply chains. Our members have risen and more harmonization among regulators of social and other inequalities that are now to the challenge of helping develop and should improve facility assessments. demanding close attention. manufacture vaccines and therapeutics for COVID-19 slowed down clinical trials Governments worldwide will be forced “The industry will view this historic Ross Burn, CEO at CatSci Future period as the catalyst that propelled us “The pharmaceutical industry will Perspectives to do things differently and collaborate look back at this time as a catalyst for in ways not seen before. We found ways change in terms of realizing the power to be more innovative because we worked of collaboration, digital transformation, In 10 years, how will the closely together to find solutions faster and redefining how quickly new vaccines industry look back on and overcome challenges at a time of and medicines can and will be developed. COVID-19? unprecedented need. This was often I hope that the incumbent mass screening achieved despite working remotely and capabilities that have proven key for many Edward Haeggström, Chief Executive conducting meetings virtually. There are countries during the pandemic will be Officer at Nanoform many lessons learned throughout this repurposed for other diseases, giving a “COVID-19 has marked a turning point experience, some of which we haven’t better outlook for patients by boosting in the pharmaceutical industry, sparking even realized today.” prevention and early detection.” one of the most efficient and collaborative global responses ever seen with the Kelly Chibale, Neville Isdell Chair in Will Downie, CEO of Vectura development and rollout of vaccines. I African-Centric Drug Discovery & “Often, out of a crisis, great things hope that, in 10 years’ time, we will Development and South Africa Research happen. This is clearly a moment in look back on this pandemic as the Chair in Drug Discovery time where the world has faced one start of a new era in the industry “COVID-19 has reminded us of of its biggest challenges, with the and a trigger to the acceleration of the importance of investing in pharmaceutical industry playing a central future developments. It showed infectious disease R&D and role in fighting the pandemic. I truly that the impossible can be also provided a blueprint believe we will look back on COVID-19 done: vaccines to the market for global collaborations as a seminal moment; a time where the from scratch on a global toward efficiently impossible was made possible. Drugs that scale and in a single year.” delivering products would normally take 10 years to bring to to address unmet market were developed in less than a year. Andrew Moore, General medical needs for the It may be the point where a seismic shift Manager at Pfizer benefit of patients.” in pharmaceutical development changed CentreOne forever. I hope so.” Andrew Moore
to explore innovative partnerships with on COVID-19 vaccines and therapies, “lab to market” journey of any therapy the private sector to address essential the research and development efforts are can be compressed like it has been for healthcare infrastructure, create viable likely to lead to a host of other discoveries, vaccines against COVID-19, then many healthcare contingency plans, and build many of which will yield significant and unmet healthcare needs can be addressed strategic reserves of key supplies. There is sustainable benefits to mankind. – and rapidly. Regulatory agencies should likely to be a reprogramming of national Scientists around the world are sharing pivot to basing their decisions to approve economic priorities toward universal information like never before and or not approve a drug on the basis of real- healthcare and providing social safety nets pharmaceutical companies are collaborating world evidence pertaining to the safety for the most vulnerable sections of society. in unprecedented ways. This kind of close and efficacy, rather than data from clinical I sincerely believe that the COVID-19 collaboration across borders is helping studies conducted under a controlled setting. experience will encourage governments science leapfrog, leading to an acceleration to engage and collectively endeavor to in drug development projects. John Chiminski deliver universal healthcare on a platform Governments worldwide will have to John Chiminski is Chair and CEO at of access and affordability. strike a better balance between regulation Catalent Pharma Solutions. In the more Despite its disruption, COVID-19 has and innovation in the interest of public than 20 years he has spent at Catalent, he also had a positive impact on medical health. Now that our priorities have radically has overseen the company’s strategy to help innovation and global collaboration in drug changed, our regulatory frameworks need accelerate the small molecule development development. We are witnessing one of to change, too. If the world can take a risk- process and improve clinical outcomes. the most frenetic and promising periods of based approach to COVID-19-related medical invention and innovation in history. treatments and vaccines, it can extend the The vertically integrated pharmaceutical Though scientists are currently focusing same approach to other diseases. If the industry of 20+ years ago would have
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During the COVID-19 response, the industry has shown tremendous The post-pandemic conversation will been challenged to mobilize capacity and innovation in technologies developed for revolve around the power of collaboration, capabilities at the scale and speed required rapid diagnostics and vaccines, and also in mRNA technology, and the regionalization/ to respond to the pandemic in the way new ways of partnering for surge capacity, localization of the supply chain. The we have, and we have seen a whole new both domestically and globally. We have collaboration between industry and level of industry collaboration. I think we also learned a lot about the global nature government created the environment have proven that CDMOs are important of supply chains. needed to accelerate vaccine development. and our customers are including us in all I think many post-COVID-19 We “cracked the code” on new tools that will manner of initiatives – for example, in discussions in industry will center on have an enormous impact on global health. COVID-19 response best practice sharing sustainability of newly established capacity, We will also be discussing in-region, for- forums. This demonstration of integration ways to integrate technologies – particularly region supply chains. Though the “Center and collaboration has helped the industry new diagnostics – into daily life, and a split of Excellence” model and one global hub solve problems and avoid many supply discussion about strengthening global worked before, the pandemic created a need chain issues, and we will do all we can to partnering and supply chains versus how for more regional supply chains. continue in this way. Regulators have also much domestic capacity a company may This past year proved we can accelerate been flexible and available, even adopting plan to build within a few countries. drug development and find ways to solve remote/on-site hybrid inspections to It will be critical to leverage the the world’s greatest health challenge when support essential products. regulatory and clinical trial efficiency we financial risk is minimized and regulatory We also learned that we can act have observed in evaluating drugs and pathways are accelerated. We must bring quickly in our industry, where timelines vaccines for the COVID-19 response. these lessons learned with us into the post- have previously been measured in years There are many examples around the world pandemic world. Historically, phases of a and decades. From contracts being put of significant improvements to reduce the clinical trial take place in sequential order in place to secure capacity, from initial development time using novel clinical trial but, with the COVID-19 vaccines, they discussion to signed agreements in weeks, designs and improving efficiency in the were done in parallel. Drug developers also not months... In one case, we went from regulatory review process. There is also the planned for commercial manufacturing tech transfer to a released batch for a high- ability to incorporate artificial intelligence early in the process. Accelerating drug profile therapeutic in just 10 days. into in silico processes that can reduce development will require parallel planning all the time for new molecule discovery or the way through commercial manufacturing. Rick Bright synthesis. This can reduce risk of candidate I’m excited about the development Rick Bright joined The Rockefeller failure – again regaining valuable time in of new modalities. It’s amazing to see Foundation in March 2021 as Senior the development timeline. years of research and development come Vice President, Pandemic Prevention & to fruition with mRNA technologies Response, Health Initiative. He will lead the Emmanuel Ligner and cell therapies finally reaching development of the Foundation’s pandemic Ligner is President and CEO at Cytiva and patients. We must continue investing in data and action platform, which will help Group Executive at Danaher Biotechnology automation and digital technologies that prevent future pandemics by identifying and Group. He has been the President and CEO will help accelerate the development and responding to the earliest alerts of a disease of Cytiva since July 2017, when it was manufacture of these novel therapeutics so outbreak and stopping it in the first 100 days. known as GE Healthcare Life Sciences. we can reach as many patients as possible. The Advanced Medicine View
How might the industry’s success in developing and rolling out COVID-19 vaccines be applied to cell and gene Miguel Forte therapy development?
Miguel Forte, CEO at Bone Therapeutics accelerate development of cell and gene chain has gained wide acceptance during “Development of vaccines for COVID-19 therapy products becomes the new normal mass vaccination campaigns. In particular, came about at lightning speed because of because there are still many urgent unmet the investment and knowledge gained the alignment of a global need, regulatory medical needs.” implementing large-scale cold chain support, and political will! Extensive logistics will carry lasting benefits for scientific collaboration and focus to Fabian Gerlinghaus, Co-Founder and future delivery of cell and gene therapies.” address a significant unmet medical need CEO Cellares is an important lesson to extrapolate “Like many, I have been fascinated by the Queenie Jang, CEO at ISCT to the field of C>, where it is key speed with which vaccine manufacturing “Looking at the variety of vaccines in use, to collaborate and combine emerging facilities can be brought online. Processes this has provided a wealth of real-world technologies to deliver what we know is for manufacturing vaccines were data to explore how differing storage, bringing enormous value to patients with transitioned from one facility to another. handling, and logistics requirements still unmanageable conditions.” Readying facilities to manufacture directly affect adoption. This has been products occurred with epic velocity. especially apparent in evaluating the Catherine Bollard, Director at the Cellares is working to accelerate time to cold chain requirements for various Children’s National Research Institute market for cell therapies in a similar way.” vaccine candidates. Although technically and Professor of Pediatrics at George possible, restrictive cold chain and product Washington University Bruce Levine, Barbara and Edward Netter characteristics (ie., ultra-low temperature “The success of the mRNA vaccine Professor in Cancer Gene Therapy at the storage, stability) will ultimately impact technology for COVID-19, I believe, has University of Pennsylvania universal adoption. Worldwide, vaccines huge implications for the cancer vaccine “Government support of vaccine that are easiest to transport, store and field and ultimately for combination research dates back years, administer will ultimately have vaccine and cell therapy approaches for and the recent influx of a higher share of the market. cancer and viral infections.” funding has spurred the Cell and gene therapy rapid development of development will need Evelina Vågesjö, Co-Founder and CEO at COVID-19 vaccines. to consider these same Ilya Pharma The success of this important market forces “Cell and gene therapies are aiming to development carries and continue to invest, cure and/or solve unmet medical needs lessons for cell and innovate, and improve currently not solved by the traditional small gene therapy in that it products to achieve molecules and biologics. The industry validates the benefit of universal adoption.” and regulators have proven that, with long-term investment in collaboration and dedication, it is possible research. Additionally, to deliver safe vaccines when needed. I the importance of hope that this level of collaboration to a robust supply
Queenie Jang Ushering in New Technology – and the Next Generation Sitting Down With… Maik Jornitz, President and CEO of G-CON Sitting Down With 51
What did you initially have planned for first: over the years, I’ve gained a great a sharpshooting approach. Overall, if we your career? deal of know-how and experience, and I look at the kind of treatments that are out Something totally different; I was actually feel I have an obligation to train the next there today, it’s clear that we are living always interested in interior design! But in generation – after all, at some point, we’ll in a much more promising era. Another those days (at least in Germany) nobody all retire! We need a solid base of next fascinating bit of rapid progress can be hired interior designers, so it wouldn’t have generation experts coming up through the found in COVID-19 vaccine development. been a good career choice! I was also very ranks to take our places. I was lucky to have We saw the industry come together and interested in biology and engineering, but an excellent mentor in sterile filtration. It is step out on thin ice by developing vaccines I didn’t know exactly how to combine the a highly critical process step. My mentor at great financial risk. I praise these vaccine two. And then I spoke to a friend of mine trained me and introduced me to other developers; it’s fantastic to see how fast who suggested bioengineering. I didn’t people – and that allowed me to learn more they’ve found a solution. even know this existed! For me, it was a and more. I want to share that knowledge match made in heaven. with the next generation to build up other If you could change one thing about the sterile filtration experts or people who industry, what would it be? How has uptake changed for ‘podular’ are passionate about the industry and, You can probably guess from my earlier cleanroom tech? ultimately, patient. I do this in many answer about change, but the one thing The early days were definitely very ways. I still write a lot of papers; some I would like to change is the adoption hard because we had to convince a very may be provocative, but this is necessary rate of new technology. If the industry conservative industry that this is a beneficial to encourage interaction and discussions. adopted new technology much faster, it tool in the toolbox. But nobody wants to be I also write books, and I give a lot of would definitely benefit patients. the first to try something new; everybody talks at conferences. Even throughout Having said that, I can also understand wants to be the fastest second. Adoption is the pandemic, I have been pre-recording why the industry is cautious – just consider becoming more rapid now – comparable to presentations for virtual conferences and the long and tedious process of post- a chain reaction. We saw the same effect taking part in Q&A sessions. approval changes. Indeed, it can take with single use technology adoption. It took three or four years to have a global change a long time to trigger, but today the majority What are the most surprising changes implemented. I think we need an evolution of applications are running with single-use you’ve seen in biopharma manufacturing – perhaps revolution – in the relationship process technologies. We’re seeing rapid over the years? between suppliers, industry, and regulators. adoption of podular cleanrooms – and When you look at the changes, they are not If new technologies lead to higher safety for the COVID-19 vaccine capacity needs surprising – other than the fact they have the product and the patient, why does it still certainly created additional realization of happened really slowly! A friend of mine take so long to implement the change? We its benefits. always says that he has spent 40 years in the need to find a way to get more technology industry and what surprises him the most is into the industry. Just think: if the Outside of G-CON, you are involved that there have been no changes! I think we semiconductor industry had not adapted, with many industry societies and have so much great technology out there, we would not have smartphones... associations. Why do you find this so but it’s implemented at such a slow pace. rewarding? When you look at other industries, like the What inspires you? There are two points to this answer. The semiconductor industry or the computer The patient, saving patients’ lives! I first reason could be considered selfish: industry, they are much faster. We need to don’t work directly on the production of I enjoy learning. I learn a tremendous be much faster and visionary to be able to therapeutics, but I’m a wheel in the clock amount from the network and peers, as serve the patient better. work. And the products we create are well as from the organization’s activities. One good surprise is the progress in cell important for production. We are still I believe that you should never stop and gene therapy. This field has become saving lives. In this industry, whatever learning. You should always be intrigued really dear to my heart and many G-CON function and whatever you do, you by something new to improve the industry projects involve cell and gene therapy. We ultimately still work for the patient. And and yourself. You need to give your input now have targeted therapies against some that is inspiring. and point of view, but also listen to cancers and rare diseases. For me, it is a real differing views from your peers. glimmer of hope. Rather than throwing This interview is available as a video at The second reason is a reflection of the a nuclear bomb at cancers, we are using https://bit.ly/350uJot
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