Hannah's Hope Fund > Research > GAN Gene Therapy

Gene Therapy


Gene Therapy

 HHF began funding the University of North Carolina at Chapel Hill Gene Therapy Center Oct. 1, 2008. Dr. Steven Gray is the lead on our GAN gene therapy project and is THE REASON we are so close to a clinical trial. Building off of 25 years of experience the Gene Therapy Center has obtained, Dr. Gray worked to develop GAN gene therapy using AAV9 viral vector that serves as a delivery truck. This "delivery truck" transports a healthy GAN gene to nerve cells, allowing the viral vector to infect the nerve cell with a healthy copy of the gene. The viral vector delivery truck we are using is called Adeno Associated Virus serotype 9. In theory, this is a one-time injection (treatment), however longevity studies are underway to fully assess whether or not a subsequent injection would be needed in the future.

 On Jan 26th, 2012, we had our Pre Investigational New Drug Meeting with the FDA. With their guidance, we have a clear path forward to a clinical trial. We need to conduct their required safety studies. The GLP toxicology studies (safety studies) are being conducted at MPI Research, in Michigan. MPI is a renowned Contract Research Organization (CRO). We will submit our IND (investigational new drug) to the FDA in the Summer of 2013, when the results are completed and assessed. They will then have 30 days to respond.

 We are working toward an April 15th deadline to submit our application to the RAC (Recombinant DNA Advisory Council), which meets quarterly.  If we have enough safety data back by the April 15th submission deadline, our meeting with the RAC will be in early June.  The 3rd regulatory hurdle we need to surpass is the Institutional Review Board (IRB) for the much awaited start of a Phase 1 clinical trial. 



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