JAG201 / Jaguar Gene Therapy - LARVOL DELTA

JAG-201-CL-101: JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Jaguar Gene Therapy, LLC | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2024 ➔ Jan 2024

Enrollment open • Trial initiation date • Gene Therapies

JAG-201-CL-101: JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency (clinicaltrials.gov) - P1/2 | N=6 | Not yet recruiting | Sponsor: Jaguar Gene Therapy, LLC

New P1/2 trial • Gene Therapies

Development of an In Vitro Potency Assay for an AAV Gene Therapy Delivering a Functional Version of SHANK3 (ASGCT 2024) - "All qualification results indicate that the assay is suitable for measuring potency to support JAG201 lot release and stability monitoring. Clinical testing of JAG201 is slated to start this year."

Gene therapy • Preclinical • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Gene Therapies • Mental Retardation • Psychiatry

Preclinical Assessment of JAG201, a Clinical Stage Gene Therapy for Severe Neurodevelopmental Disorders Caused by Mutations or Deletions in SHANK3 Including Phelan-McDermid Syndrome (PMS) and Autism Spectrum Disorder (ASD) (ASGCT 2024) - "These data demonstrate therapeutic benefit of ICV administered JAG201 on established neurobehavioral and synaptic physiological phenotypes in an animal model of Shank3 deficiency. Unilateral ICV administration of JAG201 also results in widespread biodistribution and persistent transgene expression throughout the CNS. A first-in-human clinical study with JAG201, an AAV9-based gene therapy targeting SHANK3 mutation- or deletion-related disorders, is slated to begin this year."

Gene therapy • Neurodevelopmental • Preclinical • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Gene Therapies • Genetic Disorders • Mental Retardation • Psychiatry • Sleep Disorder

Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome (Businesswire) - "Jaguar Gene Therapy...today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS)....Preclinical studies in rodents and non-human primates (NHP) demonstrated that functional SHANK3 delivery leads to improvements in neurobehavioral, cognitive and motor function abnormalities. The company plans to initiate a Phase I trial in adults with ASD or PMS where a SHANK3 mutation or deletion is present in the United States in the second half of the year."

IND • New P1 trial • Preclinical • Autism Spectrum Disorder