Story excerpt provided by Yahoo Finance.
Abeona Therapeutics, Inc. (ABEO), a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, today announced the Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH) for patients with Sanfilippo syndrome type A (MPS IIIA) or type B (MPS IIIB). Additionally, the Comite Etico De Investigacion Clinica de Euskadi (CEIC-E) has approved the ethical committee regulatory filings for both ABO-101 and ABO-102. The Company plans to file CTAs for both programs shortly for the upcoming clinical studies to be conducted at Cruces University Hospital (Bilbao, Spain).
“The GMO and Ethical committee approvals — which are similar to the Recombinant DNA Advisory Committee (RAC) and hospital Institutional Review Board processes in the United States — support our planned path to clinical trials in the EU for the treatment of Sanfilippo syndromes type A and B. Pre-clinical studies have demonstrated that a single intravenous injection of ABO-101 or ABO-102 restored the enzyme activity in the cerebral spinal fluid and serum, and also corrected the lysosomal storage pathology throughout the CNS and body,” noted Tim Miller, PhD, President & CEO. “We look forward to advancing these potentially life-changing therapies into global clinical trials and grateful for the support and guidance from regulators, clinicians, and patient community.”
Click here to read the complete article.
Originally published January 11, 2016.