Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA

Abeona Therapeutics (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, today announced updated clinical data from the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH), the company’s investigational gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.

As quoted in the press release:

“MPS IIIA is a profound and deadly lysosomal storage disease with no approved treatments available. The encouraging clinical data reported today provide strong additional support for a whole-body treatment approach involving intravenous delivery of an AAV to drive expression of the SGSH enzyme in all organs of the body, with an emphasis on expression in the central nervous system,” stated Kevin M. Flanigan, M.D., principal investigator of the trial, director of the Center for Gene Therapy at Nationwide Children’s Hospital and professor of pediatrics and neurology at The Ohio State University College of Medicine.

Click here to read the full press release.