Abeona Therapeutics enrolls high dose subject in Sanfilippo clinical trial

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Abeona Therapeutics enrolls high dose subject in Sanfilippo clinical trial

  • ABO-102, the leading clinical gene therapy program for Sanfilippo syndrome type A patients, has demonstrated central nervous system (CNS) and peripheral organ disease biopotency
  • First high dose cohort patient is enrolled, and all patients (n = 4) have cumulative 644 days post-injection with no Serious Adverse Events (SAEs) reported to date
  • Global ABO-102 enrollments in Europe and Australia commencing in the second quarter

Abeona Therapeutics Inc., a US based biopharmaceutical company, announced that the first high-dose subject was enrolled in the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH). The first-in-man clinical trial uses a single intravenous injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo syndrome type A), a devastating lysosomal storage disease that affects every cell and organ resulting in neurocognitive decline, speech loss, loss of mobility, and premature death.

“The encouraging clinical data from the low-dose patients continue to support a whole-body treatment approach using an intravenously delivered AAV to deliver and drive expression of the SGSH enzyme in all organs of the body, particularly the brain,”

stated Kevin M. Flanigan, MD, principal investigator of the clinical trial, Director of the Center for Gene Therapy and Neuromuscular Disorders Program at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine.

“Additionally, we are encouraged that the 6-month biopotency in two initial subjects are suggestive of sustained disease modification, and we look forward to presenting additional data at the WORLDSymposium lysosomal storage conference later this month.”

The ongoing Phase 1/2 clinical trial, which has received FastTrack designation by the FDA (US Food and Drug Administration), is designed to evaluate safety and preliminary indications of efficacy of ABO-102 in patients suffering from MPS IIIA. Per the design of the trial, subjects in the low-dose and high-dose cohorts received a single, intravenous injection of ABO-102 to deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease, particularly the CNS. Subjects are evaluated at multiple time points over the initial 6-months post-injection for safety assessments and initial signals of biopotency.

Previously announced 30-day post-injection data for the low dose cohort indicated:

  • ABO-102 reduced GAG (heparan sulfate) in urine 57.6% +/- 8.2%
  • ABO-102 reduced GAG (heparan sulfate) in the CSF 25.6% +/- 0.8%
  • Reduction in liver volume of 17.7% +/- 1.9%
  • Reduction in spleen volume of 17.6% +/- 7.1%



“The combination of CSF and urinary heparan sulfate GAG reduction, liver and spleen volume reduction, and neurological effects support our world leading gene therapy treatment paradigms for patients with MPS III,”

stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics.

“We look forward to Dr. Flanigan’s presentation of the biopotency and neurological data at the upcoming WORLDSymposium.”

Abeona’s MPS IIIA program, ABO-102, has been granted Orphan Product Designation in the USA and Europe, and has also received the Rare Pediatric Disease Designation in the United States.

Read the full press release from Abeona Therapeutics or visit their website for more information.