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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. His dedication is unparalleled.

The FDA prefers to have us refer to gene therapy as gene “delivery”. This is because we will not know if it’s therapeutic until it’s gone thru an efficacy trial. A phase 1 is for safety only.

With gene delivery, scientists use a benign viral vector to transport healthy genes to targeted cells. In the case of GAN, 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 (AAV). In theory, this is a one-time injection. The hope is that the delivered GAN gene will have life-long expression of the mission gigaxonin protein. 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).

When we obtain the results of the safety studies, we will report back to the FDA and they will then have 30 days to respond to us. If the safety studies go as planned, and we get the green light from the FDA and the UNC Institutional Review Board (IRB) and Bioethics Committee, we hope to begin a trial the Fall of 2013. Hold on families… we are working so hard to make this trial happen ASAP!


All website content is the opinion and observation of parents, not medical professionals. Please consult your medical doctor for medical advise.