Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten 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 European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation (EMA/OD/013/18) for Abeona’s gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children.  ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease.

As quoted in the press release:

“This designation is an important step in our CLN1 program as it encourages us to proceed towards clinical development,”  stated Carsten Thiel, Ph.D, CEO of Abeona Therapeutics Inc. “Batten disease, particularly CLN1 disease, is a severely debilitating disorder where patients do not currently have adequate therapeutic options.  Compelling pre-clinical data underscore the viability of ABO-202 as a potential treatment for CLN1 disease in humans. We remain committed to our vision of developing transformational gene therapies for patients suffering from devastating rare diseases.”

ABO-202, developed with Steven J. Gray, Ph.D, Associate Professor, Department of Pediatrics, UT Southwestern and the support of Garrett the Grand Batten Fighter, The Saoirse Foundation, Taylor’s Tale, Hayden’s Batten Disease Foundation, and the Batten Disease Support and Research Association, is anticipated to enter clinical trials in 2018.

Click here to read the full press release.