Abeona Therapeutics receives orphan drug designation in the European Union for ABO-201 gene therapy program in juvenile Batten disease.
Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing 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 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 European Union (EU) orphan drug designation for ABO-201 provides Abeona 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 recently 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,” Miller added. “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 recently published in the Journal of Neuroscience (doi: 10.1523/JNEUROSCI.1635-16.2016). www.abeonatherapeutics.com.
Filed Under: Drug Discovery