Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
JAG201 · 1 trial · 2 indications
Incidence, type, severity, and frequency of AEs
Incidence, type, severity, and frequency of SAEs
Changes in clinically significant abnormalities in laboratory values
Incidence of anti-AAV9 antibodies, anti-transgene antibodies, and T-cell reactivity to transgene over time
| Arm | Type | Description |
|---|---|---|
| Pediatric Cohort 1 | EXPERIMENTAL | Starting Dose |
| Pediatric Cohort 2 | EXPERIMENTAL | Escalated Dose |
| Name | Type | Description |
|---|---|---|
| JAG201 | GENETIC | Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3) |
Key Inclusion Criteria: 1. Is male or female, and 2 to 9 years of age at the time of JAG201 administration 2. Has a molecular confirmation of a loss of function mutation in SHANK3 or a 22q13.3 deletion classified as a Class I deletion 3. Has evidence of developmental/cognitive delay of at least 2 s...
JAG201 is an investigational gene therapy being developed for SHANK3 haploinsufficiency, a rare genetic condition that includes Phelan-McDermid syndrome. It is intended for children and adults with this disorder, which is characterized by developmental delays, intellectual disability, and other neurological symptoms.
JAG201 targets the SHANK3 gene, which is haploinsufficient in the condition it treats. The therapy is designed to address the underlying genetic cause by delivering a functional copy of the SHANK3 gene to patients, potentially restoring normal protein function and alleviating symptoms associated with SHANK3 haploinsufficiency.
JAG201 is being developed by Jaguar Health, Inc., a biopharmaceutical company traded on NASDAQ under the ticker JAGX. The company is conducting clinical research on this gene therapy candidate for the treatment of SHANK3 haploinsufficiency.
JAG201 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The ongoing Phase 1 study is actively recruiting participants to evaluate the safety and tolerability of the therapy in patients with SHANK3 haploinsufficiency.
JAG201 is being studied in a Phase 1 clinical trial registered as NCT06662188, titled 'JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency.' This single-center trial in the United States is recruiting up to 6 participants aged 2 years and older, with no healthy volunteers.
JAG201 is being investigated for SHANK3 haploinsufficiency, which is the genetic cause of Phelan-McDermid syndrome. The ongoing clinical trial includes patients with this syndrome, so JAG201 is a potential treatment for Phelan-McDermid syndrome, though it remains investigational and is not yet approved.