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Jaguar Gene Therapy to Kick off Phase I Trial of Autism, Phelan-McDermid Syndrome Gene Therapy

NEW YORK – Jaguar Gene Therapy on Tuesday said it is preparing to begin a Phase I clinical trial testing its gene therapy candidate JAG201 to treat patients with a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome.

Lake Forest, Illinois-based Jaguar said it plans to dose the first patient in Q1 2025 as part of a pediatric cohort of patients at least 2 years old, after which it will expand into adults. The firm said it has received responses from a Type C meeting with the US Food and Drug Administration, in which the FDA cleared its plans to administer the gene therapy to pediatric and adult patients.

"Our preclinical data suggest that the administration of the gene therapy early in life provides a clear potential for benefits to be realized," Jaguar CEO Joe Nolan said in a statement. "Our hope is that potential early success in the pediatric population will open the door to evaluating JAG201 in broader patient populations."

JAG201 is a gene replacement therapy being developed as a treatment for a genetic form of ASD caused by certain mutations in the SHANK3 gene and for Phelan-McDermid syndrome, a rare disorder caused by deletion near the end of chromosome 22, which includes SHANK3, and leads to symptoms like developmental delay and intellectual disability.

The one-time gene therapy aims to deliver a functional SHANK3 minigene using an adeno-associated virus serotype 9 vector, targeting neurons in the central nervous system.

An estimated 46,000 patients in the US have ASD caused by SHANK3 haploinsufficiency or Phelan-McDermid syndrome, for which there are no FDA-approved treatments on the market. The FDA has granted JAG201 rare pediatric disease and fast-track designations.

Jaguar exclusively licensed the JAG201 development program from the Broad Institute of MIT and Harvard.