Abeona Therapeutics (ABEO) Announces EU Orphan Drug Designation for ABO-102 Gene Therapy in Sanfilippo Syndrome Type A

Abeona Therapeutics (ABEO) Updates on ABO-102 Phase 1/2 MPS IIIA; Significant Avg. GAG Reduction Noted

Abeona Therapeutics Inc. (Nasdaq: ABEO) provided at the Orphan Drugs & Rare Disease Conference (London, UK), an update on clinical results through 30 days post-injection for the completed low-dose cohort (n=3) in the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH). The first-in-man clinical trial utilizes a single intravenous injection of AAV gene therapy for subjects with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease affecting every cell and organ in the body causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.

The ongoing Phase 1/2 study is designed to evaluate... More