GAN Gene TherapyGene Therapy

HHF began funding the University of North Carolina at Chapel Hill Gene Therapy Center October 1, 2008. Dr. Steven Gray is the lead on our GAN gene therapy project and is THE REASON that a clinical trial is underway. Building on the work of the Gene Therapy Center, Dr. Gray worked to develop GAN gene therapy using the 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 January 26th, 2012, we had our Pre-Investigational New Drug Meeting with the FDA Center for Biologics Evaluation and Review (CBER). With their guidance, we had a clear path forward to a clinical trial. While the Institutional Review Board (IRB) process took far too long, we are happy to announce that the first GAN patient received gene therapy to the spinal cord and brain on May 27th, 2015.

How does a gene therapy virus deliver a healthy gene to the nucleus of cells?

The virus will first bind to the outside of the cell. The surface of the cell will form a pit around the virus and basically engulf it into a small round sac that is carried from the outside of the cell to the center where the nucleus is. The AAV will break out of that sac near the nucleus and then move into the nucleus through a small pore. Once it is inside the nucleus the capsid shell will fall apart, and the DNA will spill out into its new home, the nucleus. The DNA will express the protein that doesn’t work in the cells of GAN patients.