Trial Profile

A Phase 1/Phase 2 Open-label Single Arm Study With Dose Escalation (Part A), and Dose Expansion (Part B) Parts to Evaluate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants 10 to 50 Years Old With Non-congenital Myotonic Dystrophy Type 1

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 04 Feb 2026

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At a glance

28 Jan 2026 Planned End Date changed from 28 Feb 2030 to 28 Feb 2029.
23 Sep 2025 According to Sanofi media release, first patient is planned for enrolment in late 2025.
23 Sep 2025 According to Sanofi media release, The US Food and Drug Administration (FDA) has granted fast track designation to SAR446268 for the treatment of non-congenital DM1 myotonic dystrophy type 1 (DM1).

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