Rare Daily Staff

Abeona Therapeutics said updated data from its phase 1/2 trial for ABO-102 demonstrate robust and durable clinical effects at various timepoints post-administration as a treatment for Sanfilippo syndrome type A or MPS III A, a rare a deadly lysosomal storage disorder without approved therapies.

Sanfilippo syndrome is caused by an SGSH enzyme deficiency that prevents people with the condition from properly breaking down a specific type of sugar molecule. As a result, waste fragments accumulate in cells, causing symptoms such as progressive dementia, aggressive behavior, hyperactivity, and seizures.

To date, 11 patients have been dosed with a single intravenous injection of ABO-102. Each subject received a single intravenous injection of the gene therapy for systemic delivery of a functional copy of the missing SGSH gene associated with onset and progression of the disease.

During an update at the 21st Annual Meeting of the American Society for Gene and Cell Therapy in Chicago the company said ABO-102 continues to demonstrate significant dose-dependent and time-dependent responses in key biomarkers through 18-months post-injection, including sustained reductions of heparan sulfate, the sugar molecule that is the hallmark of MPS IIIA, in the cerebral spinal fluid and urine.

“Children with MPS IIIA experience devastating quality of life consequences including neurocognitive decline, speech and mobility loss, and premature death,” said Carsten Thiel, CEO of Abeona. “As a leader in gene therapy for MPS IIIA patients, we feel encouraged by the strong data demonstrated thus far in this trial, showing significant dose- and time-dependent improvement of the underlying disease pathology.”

The U.S. Food and Drug Administration granted ABO-102 Regenerative Medicine Advanced Therapy, Rare Pediatric Disease, and Fast Track designations in the United States, and Orphan Product Designation. The European Union has also granted ABO-102 Orphan Product designation.

May 21, 2018
Photo: Carsten Thiel, CEO of Abeona

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