Abeona Therapeutics Receives FDA Orphan Drug Designation for ABO-202 Gene Therapy Program

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 Orphan Drug Designation (ODD) to its ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of infantile Batten disease.

As quoted in the press release:

“This designation builds on our clinical portfolio of AAV gene therapies that have received FDA and EMA orphan drug designations, which is an important validation of the scientific and clinical translation of these products for the severely underserved CLN1 patient population,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “The ABO-202 preclinical data from Dr. Steven Gray’s lab support the clinical translation for patients with infantile Batten disease, and provide valuable insight for potentially improving efficacy using a combination of delivery routes for CNS and whole-body benefit to remove the underlying pathology associated with the disease.  This designation helps advance the ABO-202 program and we look forward to initiating human clinical trials later this year.”

Click here to read the full press release.