Jaguar Gene Therapy Updates: JAG201 Trial Progress & Caregiver Milestone Study
CureSHANK is proud to share the latest news from Jaguar Gene Therapy on promising advancements for the Phelan-McDermid syndrome community. This includes an important update on trial progress for the JAG201 Gene Therapy Study and new eligibility criteria for the PMS Caregiver Milestone Study.
JAG201 Gene Therapy Study: Trial Progress Update
The first in-human clinical trial evaluating the safety, tolerability, and clinical activity of JAG201 continues to progress. As of July 2026, seven pediatric patients have now been dosed with JAG 201 across the three trial sites at Seaver Autism Center at Mount Sinai, Rush University, and Boston Children’s Hospital. No treatment-related serious adverse events (SAEs) have been reported. Early indications of clinical benefit have been observed across neurodevelopmental domains, including communication, motor, cognitive, and social skills.
For detailed questions about the trial, including eligibility and enrollment, please visit this website, download the FAQs, or direct inquiries to medinfo@jaguargenetherapy.com.
Study Details: The JAG201 Gene Therapy Study is an interventional trial that is enrolling children aged 2-9 years old. Adult cohorts may be initiated at a later timepoint in the study.
PMS Caregiver Milestone Study: Improve Understanding & Inform Future Research
In partnership with Psychlomere, Jaguar Gene Therapy has launched a PMS Caregiver Milestone Survey. This fully remote, online study is actively seeking caregivers of individuals living with PMS to share insights about developmental milestones. Learn more about the study and access FAQs here.
Why does this study matter?
Understanding how skills are gained, lost, or regained over time in individuals living with PMS helps researchers better understand PMS. The input from this study can provide a critical reference point for evaluating the benefits of treatments like JAG201 in clinical trials.
You May Qualify If:
You are an adult and are a caregiver of someone with Phelan-McDermid syndrome
You are willing to complete an online survey at three time points over the next 12 months
You are willing to share developmental milestone information and documentation when available
Your loved one is between ages 2 and 30
You can complete surveys in English
Final eligibility will be determined by the study team based on study requirements. Learn more about the study and check your eligibility: www.pmscaregiverstudy.com
For more information on the JAG201 Gene Therapy Study and Caregiver Milestone Study, download the FAQs here.
CureSHANK is proud to partner with and support Jaguar Gene Therapy in all of their work to advance research and treatments for individuals impacted by Phelan-McDermid Syndrome.
