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.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

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.

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

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

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.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.