Derisking orphan drug programs in 2020 and beyond

Developing therapies for rare diseases has always come with a unique set of challenges. Now, a pandemic has dramatically shifted the attention of the industry and introduced new hurdles - forcing orphan developers into a critical re-examination of their go-to-market strategies. In this webinar, Endpoints convenes four orphan drug developers who will share what they've learned so far and detail how they plan to fortify their programs. We'll also release results of our Orphan Drug Survey, which will provide additional insights from hundreds of Endpoints readers at orphan drug companies.

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Cornell Stamoran

Cornell Stamoran, Ph.D.

Vice President, Strategy & Govt. Affairs, Catalent, Inc. Founder & Co-Chair, Catalent Applied Drug Delivery Institute

Cornell Stamoran serves as Vice President of Strategy and Government Affairs for Catalent Pharma Solutions, and is a founder and Chair of Catalent's Applied Drug Delivery Institute. Cornell has worked in drug delivery for nearly 30 years, spanning oral, injectable, respiratory, ophthalmic and other routes of delivery for drugs, biologics, cell and gene therapies, and consumer health products. Cornell was recognized as a top Industry Influencer in Medicine Maker magazine's 2018 and 2019 Power Lists.

Cornell is a trustee of the Pharma/Biopharma Outsourcing Association; serves as treasurer on the board and chairs the Industry Advisory Group of the Controlled Release Society; is an Inspiring Notes editor for Drug Delivery and Translational Research, and serves on the Editorial Advisory Board of Drug Development and Delivery magazine and for several key industry conferences. Cornell has published more than two dozen articles on drug delivery, patient-focused drug design, and pharmaceutical outsourcing, and frequently speaks and posts on these topics. Cornell also served on the cross-Industry teams which negotiated GDUFA II and OMUFA I with the FDA, and will be part of the GDUFA III team.

Dr. Dietrich Stephan

Dr. Dietrich Stephan

Chairman, Chief Executive Officer, Founder, Neubase Therapeutics

Dr. Stephan is an industry veteran who is considered one of the fathers of the field of precision medicine, having trained with the leadership of the Human Genome Project at the NIH and then going on to lead discovery research at the Translational Genomics Research Institute and serve as professor and chairman of the Department of Human Genetics at the University of Pittsburgh. Stephan has identified the molecular basis of dozens of genetic diseases and published extensively in journals such as Science, the New England Journal of Medicine, Nature Genetics, PNAS and Cell. In parallel, Dr. Stephan has founded or co-founded 14 biotechnology companies and has advised an additional 12 companies. These companies are backed by top-tier investors such as Sequoia Capital, KPCB, Thiel Capital, and Khosla Ventures as well as corporate partners such as Life Technologies, Pfizer, and Mayo Clinic. Dr. Stephan received his Ph.D. from the University of Pittsburgh and his B.S. from Carnegie Mellon University.

Eric Green

Eric Green

Senior VP and General Manager TTR, Alnylam

Eric Green joined Alnylam in 2015 and is currently Senior Vice President and General Manager of the TTR Program, comprising ONPATTRO (patisiran) and vutrisiran. Both products aim to treat ATTR amyloidosis, an orphan disease. He has led the TTR Program through pivotal clinical development, global regulatory submissions and ongoing global commercial launches. Previously, Eric worked at Synageva BioPharma, where he was Vice President of Program & Alliance Management. In that role he was responsible for the oversight, management, and leadership of the company’s lead compound being developed as an enzyme replacement therapy for an ultra-orphan genetic disease. Prior to Synageva, he worked in roles of increasing scope and responsibility at Infinity Pharmaceuticals and at Genzyme Corporation. Eric received a Bachelors in Chemical Engineering from the University of Michigan, a Masters in Chemical Engineering from the MIT, and a MBA from the MIT Sloan School of Management.

Kinnari Patel, PharmD, MBA

Kinnari Patel, PharmD, MBA

COO and EVP, Development at Rocket Pharmaceuticals

Kinnari has fifteen years of rare disease Research & Development experience including in Regulatory Science, Pharmacovigilance, Policy and Quality Compliance gained at leading pharmaceutical companies including AstraZeneca, Bristol-Myers Squibb, Novartis, Hoffmann La-Roche and Pfizer. She has led small molecule and biologic development programs from Phase I through Phase IV across multiple therapeutic areas including immuno-oncology, oncology, respiratory, virology, transplantation and metabolism. Most notably, Kinnari worked on Opdivo (nivolumab) from Phase I through BLA filing for metastatic melanoma, RCC and NSCLC, submitted and gained approval of Myalept (metreleptin) for ultra-rare lipodystrophy disease, and submitted Valcyte® (valganciclovir hydrochloride) NDA for prevention of cytomegalovirus disease in pediatric kidney and heart transplant patients.

Kinnari received a B.S. in Biology and Doctorate of Pharmacy from the USciences in Philadelphia, PA and her Executive MBA from NYU Stern School of Business. Most recently, she graduated from the C-Suite Harvard Business School Advanced Management Program.

Arsalan Arif

arsalan arif

Founder & CEO, Endpoints News

Arsalan Arif is a news media entrepreneur who set out in 2015 to build his vision of an independent biotech news company at Endpoints News.