Posted on: 19 May 2016, source: Abeona Therapeutics
Cleveland-based Abeona Therapeutics has dosed its first patient in a Phase 1/ 2 Trial with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (a/k/a Mucopolysaccharidosis Type IIIA or MPS IIIA). ABO-12, a single intravenous injection, is an adeno-associated viral (AAV)-based gene therapy to treat the central nervous system and the peripheral disease manifestations in Sanfilippo patients, representing a new approach to addressing the progressive disease which often prevents children from reaching adulthood. The drug is based on more than 18 years of research and development. There are currently no approved treatments available for Sanfilippo syndrome type A.