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2nd Gene Therapy for Blood Disorders

2nd Gene Therapy for Blood Disorders


Date of beginning

Tuesday, 09 March 2021


3 days




Ellie Saunders


This email address is being protected from spambots. You need JavaScript enabled to view it.


2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape is progressing quickly with Pfizer and Sangamo kicking off their phase 3 and bluebird bio receiving European approval for their beta-thalassemia candidate. With pipelines advancing and emerging companies joining the blood disorders field, it is essential for gene therapy developers to communicate and share lessons learned to accelerate their programs to market. The 2nd Gene Therapy for Blood Disorders meeting is taking place in March 2021, focusing on the clinical and commercial challenges of developing gene therapies targeting hemophilia, sickle cell disease and beta thalassemia. Evaluate the best strategies for a commercial launch in blood disease and optimize your trial design to ultimately transform your clinical candidates into a commercial reality. On Tuesday March 09, 2021 at 8:00 am (ends Thursday March 11, 2021 at 6:00 pm) Prices: Conference Only Industry - Early Rate: USD 1499.00,Conference Only Industry - Standard Rate: USD 1999.00,Conference Only Academic - Early Rate: USD 1199.00,Conference Only Academic - Standard Rate: USD 1799.00 URLs: Tickets: Brochure: Speakers: Gabriela Denning, Vice President, Research and Development, Expression Therapeutics, Clark Paramore, Head of Value Demonstration, Vice President HEOR, bluebird bio, David Page, National Director of Health Policy, Canadian Hemophilia Society, Ian Winburn, Vice President, Global Medical Lead, Hemophilia, Endocrine and IEM, Rare Diseases, Pfizer, Julian Down, Senior Director, Gene Therapy, Poseida Therapeutics, Kate Zhang, Vice President, Editas Medicine, Lesha Shah, Assistant Professor, Medical Director, Child, Adolescent and Family Services, Icahn School of Medicine at Mount Sinai, Mount Sinai Health System, John Pasi, Professor, Haemostasis and Thrombosis, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, Mark Trusheim, Strategic Director, NEWDIGS and Visiting Scientist, MIT Sloan, Marcus Droege, Vice President and Head Global RWE, bluebird bio, Nick Li Senior Director, Health Economics and Outcomes Research, UniQure, Nicolas Garnier, Director, Patient Advocacy, Pfizer, Paul Monahan, Clinical Development Lead, Hematology, Spark Therapeutics, Richard McFarland, President, Standards Coordinating Body (SCB), Sandeep Soni, Executive Director, Clinical Development, CRISPR Therapeutics, Inc., Pablo Rendo, Global Project Head, Red Blood Disorders and Rare Disease Gene Therapy, Sanofi, Matthew Birmingham, Senior Director, Supply Chain, bluebird bio, Steven Arkin, Executive Director, Clinical Research, Rare Diseases Research Unit, Pfizer Worldwide R and D, Pfizer, Wendy Pang, Executive Director, Research and Clinical Development, Jasper Therapeutics