Ultragenyx Acquires AAV gene therapy from Abeona Therapeutics Inc
US based Biopharma Company, Ultragenyx has announced the acquisition AAV Gene Therapy (ABO-102) for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics.
ABO-102 (now UX111), is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). This therapy is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. It is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline.
The ABO-102 program has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., and PRIME and Orphan medicinal product designations in the EU.
According to the terms of the licensee agreement, Ultragenyx will assume responsibility for the ABO-102 program and in return Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments following regulatory approval.
About Ultragenyx Pharmaceutical Inc
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has a diverse portfolio of approved medicines and treatment candidates to address diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need.