Abeona Therapeutics announces top-line one year data from ABO-102 MPS IIIA trial
Abeona Therapeutics announced one year data from Cohort 1 of the ongoing ABO-102 Phase 1/2 trial for Sanfilippo syndrome Type A, or MPS IIIA. The results demonstrated robust and durable clinical effects achieved one year post-administration, with significant reductions in biopotency and biophysical measures, preservation of deep brain architecture, and stabilization across multiple neurocognitive assessments reported in comparison to untreated control subjects. The ongoing gene therapy trial for ABO-102, or AAV-SGSH, is utilizing a single intravenous injection for treatment of subjects with Sanfilippo syndrome Type A, or MPS IIIA, a rare autosomal-recessive lysosomal storage disease. Results were announced during the Alliance for Regenerative Medicine's Cell & Gene Meeting on the Mesa being held this week in La Jolla, CA. Per the design of the clinical trial, subjects received a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects were evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity. Results indicate that ABO-102 successfully reached target tissues throughout the body, including in the central nervous system.