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