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SAR446268 · 1 trial · 1 indication
Number of TEAEs post-SAR446268 administration
| Arm | Type | Description |
|---|---|---|
| SAR446268 | EXPERIMENTAL | Participants will receive a single dose of SAR446268 on Day 1 |
| Name | Type | Description |
|---|---|---|
| SAR446268 | BIOLOGICAL | Pharmaceutical form: Solution for infusion; Route of administration: IV infusion |
Inclusion Criteria: Participants are eligible to be included in the study only if all of the following criteria apply: * For Part A, participants must be 18 to 55 years of age inclusive, at the time of signing the informed consent. * For Part B, participants must be as follows: * 10 to 17 years...
SAR446268 is an investigational gene therapy being developed by Sanofi for myotonic dystrophy. It is designed to deliver an adeno-associated viral vector and is currently in Phase 1 clinical development. It has received Fast Track and Orphan Drug designations from the FDA.
SAR446268 is being studied for the treatment of non-congenital myotonic dystrophy type 1 in participants aged 10 to 55 years. The Phase 1 trial is evaluating its safety, tolerability, and efficacy in this patient population. It is not yet approved for any indication.
SAR446268 targets DMPK, the gene associated with myotonic dystrophy type 1. It is an adeno-associated viral vector-mediated gene therapy, meaning it uses a viral vector to deliver genetic material aimed at the DMPK target. This gene-focused approach distinguishes it from conventional small molecule or antibody therapies.
SAR446268 is being developed by Sanofi, which trades under the ticker SNY. Sanofi is running the clinical program for this gene therapy candidate in myotonic dystrophy type 1. The company holds both Fast Track and Orphan Drug designations from the FDA for this asset.
SAR446268 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The single active trial is currently recruiting participants, and no trials have been completed to date.
SAR446268 is being evaluated in one clinical trial, NCT06844214, titled a study of SAR446268, an adeno-associated viral vector-mediated gene therapy, in participants aged 10 to 55 with non-congenital myotonic dystrophy type 1. The trial is Phase 1, currently recruiting, with an planned enrollment of 32 participants across the United States, Argentina, Australia, Canada, Israel, and the United Kingdom.