Ultragenyx Gains Rights to Abeona Therapeutics’ Gene Therapy for Rare Disease

Ultragenyx Pharmaceutical is acquiring exclusive global rights to Abeona Therapeutics’ investigational adeno-associated virus (AAV) gene therapy ABO-102.

The gene therapy is being evaluated as a potential treatment for patients with Sanfilippo syndrome type A, a rare inherited disorder that makes children unable to break down certain carbohydrates. The disease causes serious effects, including hearing loss, speech delay and a decline in cognitive skills.

ABO-102 is administered as a one-time intravenous infusion that delivers a functional copy of the N-sulfoglucosamine sulfohydrolase gene to cells of the central nervous system and peripheral organs. It is currently being investigated in an early-stage trial.

The financial details of the licensing deal were not disclosed.