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2020: Growth & Resilience in Regenerative Medicine

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2020: Growth & Resilience in Regenerative Medicine 2020: Growth & Resilience in Regenerative Medicine 2020 ANNUAL REPORT Table of Contents The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory and reimbursement ARM Annual Report initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. 2 Letter from the CEO 24 ARM Advances Alongside Maturing Sector Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many 4 Investor Perspective 25 Virtual Programming of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative 5 Record Financings Bolster Maturing Sector 26 Capacity Building medicines and advanced therapies. 10 Real Life Miracles: The Price Family 26 What’s Next? In its 11-year history, ARM has become the global voice of the sector, representing the interests of 380+ members worldwide, including small and large companies, 12 academic research institutions, major medical centers and patient groups. The View From Science & Industry Affairs 27 Parting Letter From ARM Chair To learn more about ARM or to become a member, visit www.alliancerm.org. 14 Progressing Through the Pipeline 28 2021 ARM Officers & Board 19 The View From Public Affairs 30 ARM Staff 20 Europe Policy & Regulatory Update 32 Upcoming Events 2021 22 U.S. Policy & Regulatory Update 1 Letter from the CEO: Growth & Resilience hen we issued the Alliance for Regenerative Medicine (ARM) 2019 Annual Report a year Beyond the new approvals, there were extraordinary scientific accomplishments and ago, we never imagined what 2020 would throw at the world, the regenerative medicine recognitions. Jennifer Doudna and Emmanuelle Charpentier won the 2020 Nobel Prize in Wsector, and ARM itself. Chemistry for their discovery of the CRISPR approach to genome editing, a technique that shows clinical promise to durably treat or cure sickle-cell disease, among other applications. We saw The sector’s — and ARM’s — accomplishments in 2020 are all-the-more remarkable amid the an increased focus on, and promising clinical data for, allogeneic cell therapies with growing backdrop of COVID-19. applications of gene-editing approaches. Induced pluripotent stem cells (iPSCs) demonstrated As the pandemic took hold, we planned for retrenchment. Instead, ARM’s membership grew by 81 positive results in the clinic, including from a first-ever application for Parkinson’s disease. organizations in 2020 to exceed 380, cementing our role as the global voice of the sector. We hired ARM and the sector also achieved notable policy milestones. Our advocacy efforts led the five staff members to serve this growing membership, with a focus on operations and advocacy. Centers for Medicare & Medicaid Services (CMS) to create a DRG for CAR-T therapies that will ARM adapted to the virtual world by converting October’s Cell & Gene Meeting on the Mesa to an ensure proper reimbursement for providers. In December, CMS also finalized a rule removing online format. Instead of convening in Carlsbad, California, nearly 1,200 participants joined from barriers to innovative payment models in Medicaid programs, a significant area of focus for ARM their homes. We also launched the Cell & Gene Webinar Series and introduced ARM Connect to and its members. And the EU Pharmaceutical Strategy, a blueprint for a years-long legislative replace canceled in-person meetings. push, recognized cell and gene therapies as “milestones of major progress” in healthcare. As we expand our advocacy work through new hires and resources, ARM will devote even more We responded to the powerful movement for racial equality by recognizing that we needed attention to advancing this maturing sector’s policy goals. to address the underrepresentation of Black employees in the regenerative medicine sector. ARM stepped into action, forming the Action for Equality Task Force which created the GROW The year was not without its setbacks or growing pains, partly a result of fast-moving science RegenMed Internship Program. At least sixteen ARM member organizations will welcome the first and constrained regulator bandwidth due to COVID-19. We saw at least six therapeutic programs class of GROW interns this summer. delayed because of CMC and manufacturing-related reasons, and another four delayed due to FDA requests for additional data. There are also significant market access challenges to This was all possible because the regenerative medicine sector in 2020 raised a record overcome as we work with policymakers to accommodate the paradigm shift that durable, and amount of funding, achieved significant new scientific and clinical milestones, and brought new possibly curative, therapies represent for healthcare systems. groundbreaking therapies to patients. These challenges, however, also signify progress. For a once aspirational sector, the future At the end of 2020, there were nearly 1,100 gene, cell, and tissue-based therapeutic developers is now. We are grateful for the support of our members — therapeutic developers, patient worldwide, an increase of about 100 developers from 2019. These developers raised $19.9 billion advocates, and medical and research institutions — and will continue to work tirelessly to ensure in financing, shattering the previous record of $13.5 billion set in 2018. that patients can access transformative, life-changing therapies. Two new therapies received approval in 2020: Orchard Therapeutics’ Libmeldy, a gene therapy to treat metachromatic leukodystrophy, or MLD, was approved by the European Medicines Agency, while Tecartus from Kite, a Gilead Company, was the first CAR-T treatment approved by the Food & Drug Administration (FDA) for relapsed or refractory mantle cell lymphoma. In February 2021, the FDA approved Breyanzi, a CAR-T therapy from Bristol Myers Squibb, to treat adults with relapsed or refractory large B-Cell lymphoma. Janet Lambert A growing pipeline of new therapies are nearing regulatory decisions, with at least three Chief Executive Officer additional decisions in the U.S. and five in Europe expected in 2021. There were 1,220 clinical Alliance for Regenerative Medicine trials ongoing at the end of 2020, with 152 of those trials in Phase 3 — supporting the FDA’s and EMA’s predictions they will approve 10-20 cell and gene therapies each year by 2025. 2 3 Investor Perspective Record Financings Bolster Maturing Sector 2020 was a pivotal year for regenerative medicine financing and progress, with the pandemic 2020 was a watershed year for the rapidly evolving regenerative medicine and advanced therapy sector. revealing the importance of cell and gene therapies and genetic medicines more broadly. Whether the These innovative therapies are demonstrating profound, durable, and potentially curative therapeutic momentum is sustained in 2021 depends on whether the interest rate environment supports capital benefits for patients with a wide array of disorders — and investors have taken note. flows. Our expectation is that even if the capital markets financing environment diminishes, large biopharma will continue to invest in or acquire disruptive technologies and support valuation. In 2021 The sector raised nearly $20B in 2020, shattering previous records despite the challenges posed by and beyond, we will continue to see investor interest in in vivo gene therapy, gene editing, and cell- the COVID-19 pandemic. The sector even exceeded the previous record of $13.5B, set in 2018, by the based therapies. Manufacturing and associated regulatory delays will continue as a gating factor due to close of the third quarter, when it had raised $15.9B. Individually, many companies were able to secure the inevitable “growing pains” as academic work is translated to production at scale. massive financing rounds, with 50 deals worth more than $100M upfront. For in vivo gene therapy, we expect progress to be driven by success in new target tissues like the heart, immune regimens, gene regulation, non-viral delivery, and synthetic biology. For example, Rocket Pharma showed one of the first gene therapy successes in the heart with positive data in Danon disease, likely driving investment interest in private companies like Tenaya Therapeutics. Total Global Regenerative Medicine Financing On gene regulation, we note MeiraGTx may in 2021 release in vivo preclinical data for its gene regulation platform, a technology that could be used to enable the body as a factory for a range of proven biologics. With non-viral gene therapy, Generation Bio’s move to $2B in valuation is occurring with private companies like enGene still poised to go public. Longer-term, we expect to see investments in synthetic biology, such as transgene engineering. $19.9B For gene editing in 2020, our positive thesis was confirmed, as (1) an ex vivo proof-of-concept emerged from CRISPR Therapeutics/Vertex Pharmaceuticals, and (2) in vivo CRISPR dosing began in Editas Medicine’s and Intellia Therapeutics’ respective trials. For 2021, we anticipate initial in vivo safety and activity data, as well as longer-term follow-up on various ex vivo and CAR-T programs, to continue to drive investor and large biopharma interest. Over the longer-term, we expect active investor interest in in vivo
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