Part 3 of Gene Therapy Trial for MPS IIIA (Sanfilippo A) Has Begun

Mathew Shanley

On Monday, Rare Disease Report covered the release of new information from Abeona Therapeutics’ gene therapy trial for patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome). One year data from Cohort 1 in the company’s 3-cohort, Phase 1/2 trial evaluating a single intravenous injection of ABO-102 (AAV-SGSH) exhibited positive responses.

Today, the company announced the enrollment and administration of ABO-102 (3 x 1013 vg/kg) of the first 2 patients in Cohort 3.

Patients with MPS IIIA are unable to break down heparin sulfate (HS), a large sugar molecule. This results in symptoms like progressive dementia, aggressive behavior, hyperactivity, and seizures. The condition is typically detected in patients around 4 years of age, and there are currently no treatment options.

The first subject in Cohort 3 was enrolled at Adelaide Women’s and Children’s Hospital in Adelaide, Australia, and through 30 days post-injection, exhibited a 76% reduction in liver volume. Additionally, ABO-102 has demonstrated significant reductions of cerebral spinal fluid (CSF) HS (66.7%) and urinary HS (92.3%) in the subject.

“We are pleased to initiate our enrollment in the ABO-102 trial,” stated Nick Smith, M.D., Ph.D, Principal Investigator and Department Head of Neurology at the Adelaide Women’s & Children’s Hospital in Australia in a press release. “MPS IIIA is a profoundly disabling and progressive neurodegenerative disease with no approved treatments available. The encouraging clinical data from all cohorts to date, continues to support a whole-body treatment approach using an intravenously delivered AAV to deliver and remove disease pathology in multiple organs of the body, particularly the brain. We are grateful to the many patient foundations and parents who have supported the research needed to advance a potential treatment for this devastating unmet medical need.”

ABO-102 is designed to induce cells in the central nervous system (CNS) and peripheral organs to produce N-sulfphoglucosamine sulphohydrolase (SGSH) via the insertion of a corrective gene via adeno-associated virus (AAV) based gene therapy.

“The recently announced one-year data on our Cohort 1 patients showed durable, time-dependent responses as measured in reductions of heparan sulfate storage pathology in the CSF and urine, reduction in liver volume, stabilization of deep brain architecture and signals of stabilization of neurocognitive decline one-year post-administration. After seeing dose-dependent improvements in Cohort 2, with ABO-102 being well tolerated to date, Abeona, together with our principal investigators, dose-escalated to potentially enhance clinical benefits and prolong durability; a decision supported by the regulatory agencies across the three countries supporting our trial,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. He continued, “ABO-102 continues to be well-tolerated at all doses at all follow up timeframes, and has enabled an accelerated enrollment schedule over the coming months. We look forward to reporting additional clinical data in the ABO-102 global trial later this year.”

For more updates from Abeona about the safety and efficacy of ABO-102 in patients with MPS IIIA, follow Rare Disease Report on Facebook and Twitter.
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