RARE Daily

Ultragenyx Acquires Global Rights to Abeona Gene Therapy for Sanfilippo Syndrome

May 17, 2022

Ultragenyx Pharmaceutical has entered into an exclusive license agreement with Abeona Therapeutics for its AAV gene therapy ABO-102 for the treatment of Sanfilippo syndrome type A.

Photo: Emil Kakkis, president and CEO of Ultragenyx

Under the terms of the agreement, Ultragenyx will assume responsibility for the ABO-102 program and in return Abeona is eligible to receive tiered royalties of up to 10 percent on net sales and commercial milestone payments following regulatory approval.

Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the central nervous system. It is characterized by rapid neurodevelopmental and physical decline, often by age three. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in the rapid health decline associated with the disorder. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities.

ABO-102 (now UX111), is a novel gene therapy in phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA). ABO-102 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. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body, which 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 United States, and PRIME and Orphan medicinal product designations in the European Union.

“Based on promising data from Abeona’s clinical program, regulatory feedback to date, and our experience developing treatments for other MPS diseases, we believe ABO-102 has the potential to be a transformative therapy for patients with MPS IIIA,” said Emil Kakkis, president and CEO of Ultragenyx. “Our team’s expertise in MPS and gene therapy clinical development makes this program a seamless integration, and it has the potential to be our first gene therapy to market. The Sanfilippo community has been waiting too long for a first treatment and we believe we can help accelerate this program.”

Abeona has completed a successful Type B meeting with the U.S. Food and Drug Administration regarding the pivotal Transpher A trial to support filing and approval for ABO-102 for the treatment of patients with MPS IIIA.

Author: Rare Daily Staff

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