6 published brand profiles in this category. Cite the individual brand profile for brand-specific facts.
Orchard Therapeutics - Orchard was founded in 2015—but our roots run deeper, going back to some of the first research and clinical development involving hematopoietic stem cell, or HSC, gene therapy. Our team has played a central role in the evolution of this technology from a promising idea to a potentially life-transforming reality. Today, we carry forward our history of visionary science to imagine new possibilities for people and families affected by genetic and other severe diseases.
Sarepta - We are focused on the development of precision genetic medicines to treat rare neuromuscular and central nervous system diseases. Learn more about Sarepta.
Shire Pharmaceuticals - Takeda is a patient-focused, R&D-driven global biopharmaceutical company committed to bringing Better Health and a Brighter Future.
Swedish Orphan Biovitrum - Sobi is an international biopharmaceutical company focused on rare and debilitating diseases covering haematology, immunology and specialty care.
Takeda Rare Diseases - Takeda is a patient-focused, R&D-driven global biopharmaceutical company committed to bringing Better Health and a Brighter Future.
Ultragenyx - Ultragenyx is focused on developing first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward.