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Abeona get FDA orphan disease tag for neurological disease med

July 9, 2017

Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that the FDA has granted Orphan Drug Designation (ODD) for Abeona’s ABO-201 program (AAV-CLN3), the AAV-based single intravenous gene therapy program for juvenile Batten disease, a fatal lysosomal storage disease of the nervous system caused by autosomal-recessive mutations in the CLN3 gene.

“Receiving FDA orphan drug designation for ABO-201 augments Abeona’s suite of regulatory achievements, providing certain benefits and incentives, including marketing exclusivity, that are strategically important from a regulatory and commercial perspective,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “The published ABO-201 preclinical data from Dr. Tammy Kielian’s lab support the clinical translation for patients with juvenile Batten disease, and demonstrated the importance of selecting the right vector and delivery route for potential CNS benefit and to remove the underlying pathology associated with the disease.  This designation helps advance the ABO-201 program and we look forward to initiating human clinical trials later this year.”

Preclinical data supporting clinical trials for ABO-201 (AAV-CLN3), the AAV-based single intravenous gene therapy program for juvenile Batten disease, (juvenile neuronal ceroid lipofuscinosis, JNCL), were published in the September 2016 issue of the Journal of Neuroscience (doi: 10.1523/JNEUROSCI.1635-16.2016). The publication article can be accessed by clicking on the following link: http://www.jneurosci.org/content/36/37/9669.short.

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