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BioTech - Rare Genetic Disorders
What is the underlying science behind clinical progress in therapies that promise a genetics-based cure for devastating rare diseases and how will this contribute to the next wave of innovations? What are the implications for continued patient access to these high-cost, high-touch 2 treatments – does the ROI calculation for rare diseases still add up?

Oct 25, 2022 11:00 AM in Eastern Time (US and Canada)

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Jan Mattsson, PhD
Chief Scientific Officer, Managing Director (Sweden), and Co-FounderJan Mattsson co-founded Albireo in 2008. He has more than 20 @Albireo Pharma Inc.
Jan Mattsson co-founded Albireo in 2008. He has more than 20 years of experience in the pharmaceutical industry, covering a range of functions including R&D, business development, R&D operations and management. Prior to co-founding Albireo, he held various project leader and management positions at AstraZeneca, primarily in gastrointestinal R&D. Dr. Mattsson holds a B.Sc. in chemistry and a Ph.D. in biochemistry from University of Gothenburg.
Alnylam Pharmaceuticals - GIVLAARI
Alnylam Pharmaceuticals - Oxulmo
ChemoCentryx Inc.
Shephard Mpofu
SVP, Chief Medical Officer @Novartis Gene Therapies
Shep oversees medical affairs for Novartis Gene Therapies’ spinal muscular atrophy treatment and leads clinical development for its pipeline of promising gene therapies for rare genetic diseases. He also serves as an extended member of Novartis’ Development Committee. Shep has deep clinical and industry experience, having served in a variety of roles at Novartis since 2006. Most recently, Shep served as Novartis’ Senior Global Program Head in the Immunology, Hepatology, and Dermatology Franchise, where he played an instrumental role in clinical development of the company’s medicines. Shep graduated with a medical degree from the Godfrey Huggins School of Medicine and received a Certificate in Clinical Development from the University of Basel. He is a board-certified rheumatologist and a Fellow of the Royal College of Physicians UK.
Regeneron Pharmaceuticals
Filip Van den Wyngaert, MSc
Global Head for TTP @Sanofi
Filip is the Global Head for TTP at Sanofi. He has previously led the Cablivi program from PhII POC into commercialization at Ablynx. Filip received his MSc from University of Antwerp as well as executed an executive development program at Vlerick Business School in Leuven, Belgium.
Alexandra Moens (Moderator)
Director, Product Marketing @H1
Alexandra Moens is a PharmD who has been part of the clinical industry for over 6 years. Her aim is to make a positive impact on patient lives by working with innovative solutions, implementing enhanced clinical strategies and increasing diversity and equity in the drug development lifecycle. Her scientific background and experience working with pharmaceutical partners hand-in-hand have positively impacted clinical teams to get new insights to successfully manage their portfolio strategy in the fast evolving clinical industry.