NEW YORK – Jaguar Gene Therapy on Wednesday said it has received permission from the US Food and Drug Administration to begin human trials of its lead candidate, an investigational treatment for a genetic form of autism spectrum disorder and Phelan-McDermid syndrome.
The Lake Forest, Illinois-based biotech firm plans to initiate a Phase I trial of the gene therapy JAG201 in the US in the second half of 2024, beginning with adult patients. The firm wants to test JAG201 as a treatment for patients with certain mutations in the SHANK3 gene, which encodes for proteins involved in the formation and functioning of synapses in the brain.
The gene therapy uses an adeno-associated virus serotype 9 to deliver a functional copy of a SHANK3 minigene to neurons in the central nervous system. It's administered through a one-time unilateral intracerebroventricular injection that targets the brain and spinal cord. Based on findings from preclinical studies in rodents and nonhuman primates, Jaguar believes it can show in clinical trials that the gene therapy can restore synaptic function, which, in turn, can improve patients' neurobehavioral, cognitive, and motor functions.
The JAG201 program is exclusively licensed from the Broad Institute based on research by Guoping Feng, a senior scientist and director of model systems and neurobiology at the Broad's Stanley Center for Psychiatric Research and an associate director of the McGovern Institute for Brain Research at MIT.