SHANK3 haploinsufficiency results in synaptic dysfunction. Credit: Kateryna Kon/Shutterstock.

Jaguar Gene Therapy has received clearance from the US Food and Drug Administration (FDA) to administer JAG201, a gene replacement therapy, to both paediatric and adult patients in a Phase I clinical trial for autism.

JAG201 targets a genetic form of autism spectrum disorder (ASD) with the presence of a SHANK3 mutation or deletion, and Phelan-McDermid syndrome.

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As part of the initial Phase I clinical trial, the company plans to dose the first paediatric patient in Q1 2025, with a subsequent expansion to adult patients.

Site initiation for the trial will commence with immediate effect.

JAG201 employs an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional SHANK3 minigene directly to neurons in the central nervous system.

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SHANK3 haploinsufficiency results in synaptic dysfunction, disrupting communication between nerve cells.

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Administered through a one-time unilateral intracerebroventricular injection, the therapy aims to restore synaptic function that is critical for neurodevelopment and the maintenance of cognitive and motor skills.

Jaguar Gene Therapy CEO Joe Nolan said: “Our preclinical data suggest that the administration of the gene therapy early in life provides a clear potential for benefits to be realised.

“Our hope is that potential early success in the pediatric population will open the door to evaluating JAG201 in broader patient populations. We look forward to continuing to work with the FDA, key opinion leaders, and advocacy organisations in our efforts to bring forward a gene therapy treatment for autism spectrum disorder due to SHANK3 haploinsufficiency and genetically confirmed Phelan-McDermid syndrome.”

JAG201 recently secured the FDA’s rare paediatric disease designation, as well as fast track designation.

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