Intravenous AAV9 Gene Therapy for MPS IIIA and MPS IIIBCure Sanfilippo Foundation awarded a grant to Abeona Therapeutics, a privately-traded company that is managing license & toxicology/drug production for the AAV9 gene therapy clinical trial at Nationwide Children’s Hospital.
Project Summary: Support of formal GMP toxicology study in preparation for human clinical trial; Support for Gene therapy Drug Production to be used in human clinical trial.
Start date: October 2014
March 2017: MPSIIIA trial was initiated in May 2016. IND has been approved for commencement of MPSIIIB gene therapy trial.