Odylia Therapeutics is a non-profit corporation working to help develop gene therapies for rare eye disease; in this clip Harrison Brown, PhD, BBA, the company’s Chief Science Officer, discusses what will be the next ‘big thing’ in gene therapy development.
Mark Pykett, PhD, Chief Scientific Officer at PTC Therapeutics, discusses three of his company’s clinical programs, including Spinal Muscular Atrophy, Familial Dysautonomia (FD) and AADC Deficiency.
Mucopolysaccharidoses (MPSs) are a group of genetic lysosomal disorders in which persons have low levels of specific enzymes that leads to an abnormal accumulation of complex carbohydrates (mucopolysaccharides or glycosaminoglycans).
John Maslowski, President and Chief Executive Officer (CEO) of Fibrocell Science, discusses his company’s gene therapy pipeline, including a therapy to treat localized scleroderma.
Scott Dorfman, CEO of Odylia Therapeutics, explains the origins and philosophy of Odylia Therapeutics, a non-profit company designed to find treatments for rare disease likely to be overlooked by for-profit organizations.
Akshay Vaishnaw, MD, PhD, Chief Medical Officer at Alnylam Pharmaceuticals discussed the company’s approved RNA interference (RNAi) drug, Patisiran, to treat to hereditary transthyretin-mediated amyloidosis (ATTR).