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Fully Human Domain Antibody Therapeutics: the Best of Both Worlds
Drug Discovery Fully Human Domain Antibody Therapeutics: The Best of Both Worlds By combining the therapeutic benefits of small molecule drugs with those of fully human antibodies, Domain Antibodies are expected to have strong therapeutic and commercial potential. By Robert Connelly at Domantis Robert Connelly is Chief Executive Officer of Domantis. He has over 22 years’ commercial experience of the life science sector, including that gained in the fields of diagnostics, drug discovery technologies and antibody therapeutics. Prior to joining Domantis, he was CEO of Veritas Pharmaceuticals (Los Angeles, USA), an in vivo imaging start-up company. He spent over five years with IGEN International, latterly as Senior Vice President and General Manager, Life Sciences, where he took part in the company’s IPO and financing rounds, raising $130 million. The first 11 years of his career were spent at Abbott Laboratories in sales, marketing and management positions. Domain Antibodies (dAbs) are the smallest functional variable regions of either the heavy (VH) or light (VL) binding units of antibodies. At Domantis, we are chains of human antibodies. Domantis scientists applying our proprietary know-how in dAbs to deliver have used the variable domains sequences of human human therapies that address large, unmet medical antibodies to create a series of large and highly needs in areas such as inflammation, cancer and functional libraries of fully human dAbs, with each autoimmune diseases. Three and a half years after library comprising at least 1010 different dAbs. The opening our laboratories, we have a dozen proprietary dAbs selected from these libraries are both specific therapeutic programmes underway, and an additional for their biological target and are well folded and eight therapeutic programmes with partners. -
Power List 2018
APRIL 2018 # 40 Editorial Upfront In My View Sitting Down With Stop and look at how far Preparing for the EU’s new What can algae teach us Sophie Kornowski-Bonnet, the industry has come data protection regulation about medicine design? Roche Partnering 09 10 – 11 20 – 21 50 – 51 100 Power List 2018 www.themedicinemaker.com Continuous Growth Fibra-Cel® disks—3-D growth matrix for perfusion and continuous processes Suspend your disbelief: > Less susceptible to shear forces, The three-dimensional Fibra-Cel matrix clogging, and fouling entraps anchorage dependent and > Ideal for secreted product and vaccine suspension cells—for optimized growth production conditions and increased yields. > Suitable for GMP production > For use in autoclavable, sterilize-in- place or BioBLU® Single-Use Vessels www.eppendorf.com/Fibra-Cel Fibra-Cel® is a registered trademark owned by Imerys Minerals California, Inc., USA and licensed to Eppendorf, Inc., USA. Eppendorf®, the Eppendorf Brand Design and BioBLU® are registered trademarks of Eppendorf AG, Germany. All rights reserved, including graphics and images. Copyright © 2018 by Eppendorf AG. tmm_epp_ad_210x266_2018_04.indd 1 28.03.18 13:34 Online this Month The Power List The 2018 Power List, starting on page 24 of this issue, features 100 of the most inspirational professionals involved in A Scientist Walks into a Bar... format to the public”. Generally, drug development. The list was compiled it involves scientists speaking on a based on reader nominations and And gives a presentation as part variety of topics, from medicine, to feedback from a judging panel – but any of Pint of Science, a global science neuroscience, to robotics and more, in list will always be subjective. -
Telaprevir (Incivek)
© Hepatitis C Online PDF created September 25, 2021, 4:19 pm Telaprevir (Incivek) Discontinued. This treatment has been discontinued. Table of Contents Telaprevir Incivek Summary Drug Summary Adverse Effects Class and Mechanism Manufacturer for United States FDA Status Indications Dosing Clinical Use Cost and Medication Access Resistance Key Drug Interactions Full Prescribing Information Figures Drug Summary Although telaprevir was a promising direct-acting antiviral agent that had impact in the hepatitis C treatment field during 2011 to 2013, it was subsequently replaced by newer direct-acting antiviral agents that were more effective, better tolerated, and more convenient. Based on the dwindling role of telaprevir after newer direct-acting antiviral agents were approved, Vertex pharmaceuticals discontinued the sales and distribution of telaprevir in the United States in October 2014. Telaprevir does have some current importance since persons who previously failed a telaprevir-based regimen may have developed resistant associated variants, which could potentially impact subsequent therapy. Adverse Effects The most significant adverse effects reported in the main registration trials and in post-marketing experience were rash, anorectal complaints, and anemia. When comparing triple therapy of telaprevir, peginterferon, and ribavirin with dual therapy of peginterferon and ribavirin alone significant differences were noted with rash (56% versus 34%), anemia (36% versus 17%), and anorectal complaints that include anorectal discomfort, anal pruritus, and hemorrhoids (29% versus 7%). In most cases, the rash that develops is eczematous or maculopapular in character and mild to moderate in severity; the rash is typically manageable with good skin care and topical emollients or corticosteroids. In some instances, however, telaprevir has caused serious skin Page 1/5 rashes, including Steven's Johnson Syndrome (SJS), Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS), and Toxic Epidermal Necrolysis (TEN). -
ARK GENOMIC REVOLUTION MULTI SECTOR ETF (ARKG) HOLDINGS As of 09/27/2021
ARK GENOMIC REVOLUTION MULTI SECTOR ETF (ARKG) HOLDINGS As of 09/27/2021 Company Ticker CUSIP Shares Market Value($) Weight(%) 1 TELADOC HEALTH INC TDOC 87918A105 3,937,797 531,208,815.30 6.99 2 EXACT SCIENCES CORP EXAS 30063P105 3,971,013 381,296,668.26 5.01 3 PACIFIC BIOSCIENCES OF CALIF PACB 69404D108 13,696,148 350,347,465.84 4.61 4 VERTEX PHARMACEUTICALS INC VRTX 92532F100 1,722,281 316,228,014.41 4.16 5 FATE THERAPEUTICS INC FATE 31189P102 4,825,395 312,926,865.75 4.12 6 IONIS PHARMACEUTICALS INC IONS 462222100 8,572,965 310,341,333.00 4.08 7 REGENERON PHARMACEUTICALS REGN 75886F107 430,742 275,201,063.80 3.62 8 TWIST BIOSCIENCE CORP TWST 90184D100 2,237,350 250,829,308.50 3.30 9 TAKEDA PHARMACEUTIC-SP ADR TAK UN 874060205 13,592,076 229,570,163.64 3.02 10 ACCOLADE INC ACCD 00437E102 5,268,242 226,850,500.52 2.98 11 INTELLIA THERAPEUTICS INC NTLA 45826J105 1,508,421 224,965,907.94 2.96 12 VEEVA SYSTEMS INC-CLASS A VEEV 922475108 741,198 222,307,516.14 2.92 13 CAREDX INC CDNA 14167L103 3,433,475 220,978,451.00 2.91 14 CRISPR THERAPEUTICS AG CRSP H17182108 1,804,041 210,044,493.63 2.76 15 INCYTE CORP INCY 45337C102 2,893,385 199,643,565.00 2.63 16 INVITAE CORP NVTA 46185L103 6,059,066 182,135,523.96 2.40 17 ADAPTIVE BIOTECHNOLOGIES ADPT 00650F109 4,888,391 178,377,387.59 2.35 18 BEAM THERAPEUTICS INC BEAM 07373V105 1,849,698 175,110,909.66 2.30 19 SIGNIFY HEALTH INC -CLASS A SGFY 82671G100 8,107,683 160,937,507.55 2.12 20 UIPATH INC - CLASS A PATH 90364P105 2,955,628 155,761,595.60 2.05 21 CASTLE BIOSCIENCES INC CSTL 14843C105 2,130,211 -
List of Section 13F Securities
List of Section 13F Securities 1st Quarter FY 2004 Copyright (c) 2004 American Bankers Association. CUSIP Numbers and descriptions are used with permission by Standard & Poors CUSIP Service Bureau, a division of The McGraw-Hill Companies, Inc. All rights reserved. No redistribution without permission from Standard & Poors CUSIP Service Bureau. Standard & Poors CUSIP Service Bureau does not guarantee the accuracy or completeness of the CUSIP Numbers and standard descriptions included herein and neither the American Bankers Association nor Standard & Poor's CUSIP Service Bureau shall be responsible for any errors, omissions or damages arising out of the use of such information. U.S. Securities and Exchange Commission OFFICIAL LIST OF SECTION 13(f) SECURITIES USER INFORMATION SHEET General This list of “Section 13(f) securities” as defined by Rule 13f-1(c) [17 CFR 240.13f-1(c)] is made available to the public pursuant to Section13 (f) (3) of the Securities Exchange Act of 1934 [15 USC 78m(f) (3)]. It is made available for use in the preparation of reports filed with the Securities and Exhange Commission pursuant to Rule 13f-1 [17 CFR 240.13f-1] under Section 13(f) of the Securities Exchange Act of 1934. An updated list is published on a quarterly basis. This list is current as of March 15, 2004, and may be relied on by institutional investment managers filing Form 13F reports for the calendar quarter ending March 31, 2004. Institutional investment managers should report holdings--number of shares and fair market value--as of the last day of the calendar quarter as required by Section 13(f)(1) and Rule 13f-1 thereunder. -
Vertex Pharmaceuticals Global Medical Affairs Pharmd Fellowship Program 2020 Message from Global Medical Affairs Leadership
Vertex Pharmaceuticals Global Medical Affairs PharmD Fellowship Program 2020 Message from Global Medical Affairs Leadership Dear Candidates, Vertex Pharmaceuticals in collaboration with Northeastern University is privileged to host and expand the PharmD Fellowship Program. We are excited to work with our fellows as integral members of our team and facilitate in-depth exposure to a range of functional areas, offering them the critical opportunity to gain extensive experience in the biopharmaceutical industry. In addition to fostering each individual’s personal and professional growth, the program’s primary goal is to extend the visibility of valuable contributions that a Doctor of Pharmacy can bring to the pharmaceutical industry. At Vertex, our Global Medical Affairs fellows are highly encouraged to take on immersive projects, allowing them to work across departments with leaders in the industry. We advocate for them to partake in truly unique, impactful work that develops their skills, sparks curiosity, and drives the science. Vertex’s open and innovative culture allows the fellows to ask questions, challenge the status quo, and explore their interests across multiple expertise areas. As a pharmacist, I am extremely proud of the establishment of the Vertex PharmD Fellowship program in Global Medical Affairs and excited to welcome our future fellows. Vertex is an ideal place for fellows to start their professional career, and we are dedicated to the development of the next generation of pharmacist leaders in the biopharmaceutical industry. As Vertex continues to grow and evolve into a company in multiple disease areas, I am confident that our fellows will be an essential part of moving the needle to deliver on our commitment to patients. -
Humankind 2.0: the Technologies of the Future 6. Biotech
Humankind 2.0: The Technologies of the Future 6. Biotech Piero Scaruffi, 2017 See http://www.scaruffi.com/singular/human20.html for the full text of this discussion A brief History of Biotech 1953: Discovery of the structure of the DNA 2 A brief History of Biotech 1969: Jon Beckwith isolates a gene 1973: Stanley Cohen and Herbert Boyer create the first recombinant DNA organism 1974: Waclaw Szybalski coins the term "synthetic biology” 1975: Paul Berg organizes the Asilomar conference on recombinant DNA 3 A brief History of Biotech 1976: Genentech is founded 1977: Fred Sanger invents a method for rapid DNA sequencing and publishes the first full DNA genome of a living being Janet Rossant creates a chimera combining two mice species 1980: Genentech’s IPO, first biotech IPO 4 A brief History of Biotech 1982: The first biotech drug, Humulin, is approved for sale (Eli Lilly + Genentech) 1983: Kary Mullis invents the polymerase chain reaction (PCR) for copying genes 1986: Leroy Hood invents a way to automate gene sequencing 1986: Mario Capecchi performs gene editing on a mouse 1990: William French Anderson’s gene therapy 1990: First baby born via PGD (Alan Handyside’s lab) 5 A brief History of Biotech 1994: FlavrSavr Tomato 1994: Maria Jasin’s homing endonucleases for genome editing 1996: Srinivasan Chandrasegaran’s ZFN method for genome editing 1996: Ian Wilmut clones the first mammal, the sheep Dolly 1997: Dennis Lo detects fetal DNA in the mother’s blood 2000: George Davey Smith introduces Mendelian randomization 6 A brief History of Biotech -
United States Securities and Exchange Commission Form
UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of The Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): June 17, 2005 VERTEX PHARMACEUTICALS INCORPORATED (Exact name of registrant as specified in its charter) MASSACHUSETTS 000-19319 04-3039129 (State or other jurisdiction of (Commission File Number) (IRS Employer Identification incorporation) No.) 130 Waverly Street Cambridge, Massachusetts 02139 (Address of principal executive offices) (Zip Code) (617) 444-6100 Registrant’s telephone number, including area code: Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below): o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) o Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) o Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) o Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) Item 8.01. Other Events. On June 17, 2005, Vertex Pharmaceuticals Incorporated and Merck & Co., Inc. issued a joint press release that announced the initiation of an additional Phase I study with VX-680, a small molecule inhibitor of Aurora kinases. A copy of that press release is attached hereto as Exhibit 99.1 and is incorporated herein by reference. Item 9.01. -
A Balanced Trade Context for HIV Patent Pool
iMedPub Journals 2011 TRANSLATIONAL BIOMEDICINE Vol. 2 No. 1:3 This article is available from: http://www.transbiomedicine.com doi: 10:3823/420 A Balanced Trade Context for HIV Patent Pool Daniele Dionisio, M.D. Member, European Parliament Working Group on Innovation, Access to Medicines and Poverty- Related Diseases. Reference Advisor for “Drugs for the developing countries”, SIMIT (Italian Abstract Society for Infectious and Tropical Diseases). Former Director, Infectious Disease Division, Pistoia city Hospital (Italy). Background: Reluctance of the multinational pharmaceutical companies to join the Medicines Patent Pool plan for HIV drugs (antiretrovirals-ARVs) might undermine E-mail: [email protected] its desirable objective of scaling up long-term, extended access to novel, affordable and appropriate ARV formulations in resource-limited settings. Methods: This paper makes an analysis of conflicting issues and calls for a trade context facilitating a reverse of multinational drug manufacturers’ reluctance to join patent pool. To this aim, partnerships between multinational companies are urged first to make cutting edge brand fixed-dose combination (FDC) ARVs promptly avail- able, and secondly, to allow patent pool agreements to be negotiated immediately afterwards. This context rejects clauses that exclude middle-income countries from sharing in the patent pool. Expected results: The suggested trade context can help speed up the partici- pation of originator pharmaceutical companies in the Medicines Patent Pool, while allowing them to maintain competitiveness, take advantage of incoming joint ven- ture opportunities and circumvent the need for additional incentives. This context potentially tackles in an appropriate way the directions of evolution in emerging markets, while bringing benefits to resource-limited populations, multinational drug corporations and manufacturers from middle-income countries. -
RNA-Based Reprogramming
RESEARCH HIGHLIGHTS STEM CELLS RNA-based reprogramming RNA molecules can both induce pluripo- RNA-based technique instead of viral tech- development, began producing myotubes. In tency and direct differentiation. niques for much of its induced pluripotent fact, Rossi expects that techniques using RNA The ability to make person-specific pluri- stem cell production. for differentiation will probably be adopted potent stem cells opens up many possibilities Getting the technique to work required more quickly than using RNA for reprogram- for research tools and perhaps even therapies. a lot of trial and error, says Rossi. His first ming. “I think people will have an easier time But most reprogramming techniques alter cells’ attempts to reprogram cells with RNA failed in differentiation because it’s often one factor genomes, making cells less predictable and more because cells responded as if they had been rather than several,” he says. prone to cancer-like growth. Methods to repro- infected by a virus, committing apoptosis or Making the modified RNA itself is easy, gram cells without changing their genomes launching other antiviral defenses. Swapping says Rossi, but researchers will need to exist but are inefficient and labor-intensive; out two nucleosides with similar analogs mit- spend some time tinkering to find the best most also rely on DNA and so still carry the igated the response and allowed the research- conditions for their systems. Every aspect risk of unintended genetic alterations. ers to reprogram cultured human cells by of culture conditions affects how cells are A reprogramming technique recently applying daily doses of RNAs coding for four transfected with RNA, he says. -
The Path Less Costly
COMMENtaRY CASE STUDY The path less costly Brady Huggett When faced with a competitive threat, two companies took diametrically opposite approaches. Both were ultimately successful, but Genzyme’s decision proved to be the cleaner and cheaper option. s the world’s leader in developing enzyme-replacement drugs, and Novartis of Basel. Houston-based Tanox was founded in 1986 to AGenzyme has always understood the importance of first to market. In focus on anti-IgE antibodies and by 1989 was looking for a clinical 1991, the company obtained approval for Ceredase (alglucerase injection), development partner; it sent samples of its candidate to both Genentech an enzyme replacement therapy for lysosomal storage disease (LSD) type and Ciba Geigy (the company that would later become Novartis). 1 Gaucher. Three years later, its second-generation product, Cerezyme Genentech passed. Ciba Geigy, however, began working with Tanox (imiglucerase for injection), was also cleared for commercialization. The on anti-IgE antibodies for allergic diseases. lack of treatments for lysosomal storage diseases and effective patient out- Yet Genentech clearly had interest in the area, because it began its own reach and marketing meant that Genzyme could command soaring prices anti-IgE program a few years later—a move that prompted a misappro- for its orphan treatments. In 2000, the two drugs alone provided 66% of priation suit from Tanox. The companies fought in court for three years Genzyme’s entire product revenue. before Genentech, Tanox and Novartis reached a settlement and entered At this time, Novazyme Pharmaceuticals was a young company devel- a cross-licensing agreement for anti-IgE antibodies. -
Empire State Stem Cell Board Full Board Committee Meeting Minutes December 17, 2010
Empire State Stem Cell Board Full Board Committee Meeting Minutes December 17, 2010 The Empire State Stem Cell Board held a meeting on Friday, December 17, 2010, at the offices of the Department of Health, 90 Church Street, New York, New York. Dr. David C. Hohn, M.D., presided as Vice Chair. Funding Committee Members Present: Dr. David Hohn, Chairperson Mr. Kenneth Adams Mr. Robin Elliott Dr. Bradford Berk* Dr. Mario Loomis * via videoconference Ms. Madelyn Wils Funding Committee Members Absent: Dr. Richard Daines Dr. Allen Spiegel Dr. Gerald Fischbach Dr. Michael Stocker Dr. Hilda Hutcherson Ethics Committee Members Present: Ms. Jann Armantrout Dr. Vivian Lee Fr. Thomas Berg Rev. H. Hugh Maynard-Reid Ms. Brooke Ellison* Dr. Samuel Packer Dr. Samuel Gorovitz Mr. Robert Swidler Dr. Robert Klitzman *via videoconference Ethics Committee Members Absent: Ms. Nancy Dubler Department of Health Staff Present: Dr. David Anders Ms. Susie Han Mr. Andrew Bentley Dr. Matthew Kohn Ms. Bonnie Brautigam Ms. Beth Roxland Dr. Kathy Chou Ms. Lakia Rucker Ms. Janet Cohn Dr. Lawrence Sturman Presenter: Dr. Derrick Rossi Observers Present: Ms. Jennifer Becht Ms. Caroline Marshall Mr. Joe Feldman Mr. David McKeon Mr. Mike Jolin Ms. Valeria Vavassori-Chen Ms. Elizabeth Misa Welcome and Introductions Dr. Hohn called the meeting to order and welcomed Board members, staff, and the public. He introduced Ms. Jann Armantrout, the newest member of the Ethics Committee. Dr. Hohn then asked members and staff to introduce themselves and provide their title and affiliation. Dr. Hohn stated that during the lunch break, several members had approached him to request special recognition of Dr.