European regulators have given Abeona Therapeutics Inc., (NASDAQ:ABEO) the green light to go on with its planned clinical trials for two potential treatments for Sanfilippo syndrome, a terminal genetic disorder. Abeona has obtained approval to proceed with its candidates AB0-101 and ABO-102. Abeona intends to carry out the trials at the Cruces University Hospital in Bilbao, Spain.
Abeona has said that the clinical studies will specifically target people with Sanfilippo syndrome type A, also known as MPS IIIA and type B, also known as MPS IIIB. The company has not indicated when it plans to begin the two clinical programs, but it noted that it will be filing CTAs for the two studies shortly. The GMO and Ethical approvals are only the initial regulatory approvals that Abeona is seeking for its programs in Europe.
The European GMO and Ethical Committees are equivalent to Recombinant DNA Advisory Committee (RAC) and Hospital Institutional Review Board in the U.S.
MPS IIIA and MPS IIIB are life-threatening genetic diseases caused by a gene defect that results in abnormal accumulation of sugars in body tissues. The company’s drug candidates use viral vectors to introduce functional copies of missing genes across the blood/brain barrier. MPS IIIA and MPS IIIB are especially prevalent in Europe and Australia. The disease has no known cure and care is largely supportive.