Preliminary Results Show Promise in Clinical Trial of ABO-101 for Sanfilippo Syndrome Type B
Abeona Therapeutics has reported preliminary 30-day safety and efficacy results from the first patient dosed in the company’s ongoing Phase 1/2 trial of a single treatment gene therapy candidate for Sanfilippo syndrome type B. The trial (NCT03315182) is testing Abeona’s first-in-human, adeno-associated viral (AAV)-based gene therapy ABO-101. The treatment…