NEWS

Dr. Henry Lee, Ph.D., Boston Children’s Hospital, F.M. Kirby Neurobiology Center joins the Journal of Inherited Metabolic Disease (JIMD) podcast. Dr. Lee discusses the challenges of developing gene therapies for inherited disorders of neurotransmission. Additionally he shares the progress that his group has made with succinic semialdehyde dehydrogenase (SSADH) deficiency.

Click here to listen to Dr. Lee’s Podcast with JIMD

From Dr. Lee’s update you can see that their gene therapy work on the mouse model has been successful, in that they can reverse the effects of SSADHD.

Wardiya Afshar-Saber, PhD., at BCH is also working on the human iPSC derived neurons in vitro with the hope of mimicking the gene therapy mouse model results.

These results will be combined to provide the foundation for the remaining steps of the Preclinical Research phase of the drug approval process for the Food and Drug Administration (FDA) in the United States. 

This is an exciting step for the SSADH Community and we are grateful to the researchers and clinicians hard work and dedication that got us to this point.

Click here to view the entire PDF “Understanding the FDA approval process for gene therapies” from Insightful Moments.