Jaguar Gene Therapy Announces Successful Completion of Dosing of First Patient Cohort in Clinical Trial Evaluating JAG201 for the Treatment of a Leading Monogenic Cause of Autism Spectrum Disorder Known as Phelan-McDermid Syndrome
Jaguar Gene Therapy, LLC (“Jaguar”) a clinical-stage biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations, today announced completion of dosing of Cohort 1 in its first-in-human clinical trial evaluating the safety, tolerability and clinical activity of JAG201 in patients with SHANK3 haploinsufficiency, a leading monogenic cause of autism spectrum disorder that is clinically diagnosed as Phelan-McDermid syndrome. The first two patients in dose-escalation Cohort 2 have been dosed, and Jaguar anticipates target enrollment and dosing to be completed in Cohort 2 in Q2 2026. No treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) have been reported in the trial to date. Early indications of clinical benefit have been observed across neurodevelopmental domains, including communicative, motor, cognitive and social.
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
Jaguar Gene Therapy, a clinical-stage biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations, today announced the receipt of U.S. Food and Drug Administration (FDA) responses from a Type C meeting regarding the Phase I clinical trial for JAG201, a gene replacement therapy that targets a genetic form of autism spectrum disorder (ASD) where a SHANK3 mutation or deletion is present, and Phelan-McDermid syndrome. Specifically, the company received the FDA’s agreement to administer JAG201 to both pediatric and adult patients. Jaguar Gene Therapy plans to dose the first pediatric patient in Q1 of 2025 with expansion into adults following the pediatric cohort.
New Resource for the Galactosemia Community Launches to Empower Families as They Navigate Lifelong Genetic Disease
The Galactosemia Foundation, a non-profit charitable organization that advocates for people with galactosemia and their families, and Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the publication of Navigating Galactosemia Life Stages: A Handbook for the Galactosemia Community. The resource, developed with input from galactosemia experts, provides education and tools to help navigate the significant lifelong challenges of Type 1 galactosemia, a rare genetic disease caused by mutations in the GALT gene that can lead to a severe deficiency in functional GALT enzyme. The handbook was created to support the galactosemia community in managing the many disease complications that can manifest throughout life.
