Support for AAV9 Gene Therapy for MPS IIIA and MPS IIIB

June 1, 2015

Cure Sanfilippo Foundation made a grant to Nationwide Children’s Hospital (Columbus, OH) to conduct gene therapy to treat Sanfilippo Type A (MPS IIIA) and Type B (MPS IIIB).

Project Summary: Funding support of staff to enable necessary clinical trial preparatory steps to move forward. The positions are 1) Genetics Clinical Research Fellow (1 year), and 2) Program Manager (2 years).

The duties of these positions include, but not limited to: analysis of the data from the natural history study; drafting of manuscripts related to it; participation in aspects of preparing for gene transfer trials; and analysis and presentation of the data derived from them, participation in the development and filing of regulatory documents, development of standard operating procedures and source documents, management of interactions with contracted services (such as the hospital’s clinical research services and outside monitoring contract researchers organizations), and interactions with the Institutional Review Board, FDA submission of IND and other needed paperwork, additional program coordination, finalizing publication of natural history study data.

Dates: June 2015 – May 2017

March 2017: MPSIIIA gene therapy clinical trial is underway. Safety has been established in the low dose cohort (3 patients) to date.  Data presented at the World Symposium in February 2017 noted a 67% decrease in spinal fluid heparan sulfate (the toxic storage material) levels, lowered urine heparan sulfate levels and continued reduced liver and spleen sizes. High dose cohort patient dosing is proceeding. The IND has been approved for commencement of MPSIIIB gene therapy trial.

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