Biotech Showcase™ 2016: Interview: Abeona breaking into rare diseases

February 4, 2016
Three-year-old Abeona Therapeutics is poised to enter the clinic with its lead gene therapy programs targeting some devastating lysosomal diseases. In collaboration with patient advocate groups, researchers and clinicians, Abeona President and CEO Tim Miller tells Informa Pharma Insights global director of content Mike Ward that the company expects to start global clinical trials in first quarter of 2016 of AB0-101 (AAV9 NAGLU) and ABO-102 (scAAV9 SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). Having raised some USD 60 million, the company is also developing ABO-201 (scAAV9 CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV LK19 FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Find more videos and articles at http://www.partnering360.com/insight For more information on Biotech Showcase™, visit our webpage: http://www.ebdgroup.com/bts/index.php
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