ResearchFocus on the pharmaceutical and biotech industry
- Next Generation Gene Therapy
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.
- PTC Therapeutics’ Clinical Focus on Rare Diseases: SMA, FD, and AADC Deficiency
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.
- Mucopolysaccharidosis (MPS) Resource Center
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).
- Gene Therapy to Treat Localized Scleroderma
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.
- Odylia Therapeutics – A Nonprofit Gene Therapy Company
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.
- Update on Patisiran to Treat Hereditary ATTR
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).
- Gene Therapy to Treat Epidermolysis Bullosa
John Maslowski is President and Chief Executive Officer (CEO) of Fibrocell Science, a biotech company that is developing cell and gene therapies for a variety of rare, mostly skin-related, conditions.
- Rare Disease Drug Development and Patient Centricity
Nick Kenny, PhD, Chief Scientific Officer at Syntheos Health, understands the important role patients play in developing orphan drugs and designing clinical trials. Recently, we talked to Dr. Kenny at the BIO International Convention held in Philadelphia, PA.
- Accelerating the Road to Treatment: The Global Commission to End the Diagnostic Odyssey for Children
The Global Commission to End the Diagnostic Odyssey for Children, co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, is a multidisciplinary group of patient advocates, physicians and other experts working together to help solve the complex challenges impacting the rare disease community.
- Grouping Sickle Cell Disease Patients by Symptoms Rather Than Ethnicity
Sickle Cell 101 is collected data to better group and quantify how patients manage their symptoms and treatment in the real world.