Abeona Therapeutics Receives FDA Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease

Abeona Therapeutics (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced today that the FDA has granted Rare Pediatric Disease Designation for the ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of CLN1 disease (infantile and late infantile onset Batten disease).

As quoted in the press release:

“This Rare Pediatric Disease designation for ABO-202 is a significant recognition of the strength of the data supporting a potential treatment for patients with CLN1, and is bolstered by the previous Orphan Drug designation from the FDA,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “These regulatory designations highlight the urgent need for a treatment for this devastating rare disease, and we look forward to initiating human clinical trials later this year.”

Click here to read the full press release.