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.

About Ultragenyx

Ultragenyx is a biopharmaceutical company involved in the Research and Development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases. Ultragenyx is based in Novato, CA and Brisbane, and has a presence in the Boston area, including a gene therapy plant under construction as of 2021. The company’s Latin American headquarters is located in Miami.

May 19, 2022


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