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SAR 446268

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Drug Profile

SAR 446268

Alternative Names: SAR-446268

Latest Information Update: 25 Sep 2025

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At a glance

  • Originator Sanofi
  • Class Gene therapies
  • Mechanism of Action Gene transference; Myotonin protein kinase expression inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Myotonic dystrophy
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Myotonic dystrophy

Most Recent Events

  • 23 Sep 2025 SAR 446268 receives Fast Track designation for Myotonic dystrophy [IV,Infusion] (In adolescents, In children, In adults) in USA
  • 23 Jul 2025 Phase-I/II clinical trials in Myotonic dystrophy (In children, In adolescence, In adults) in USA, Argentina (IV) (NCT06844214)
  • 25 Mar 2025 Preclinical trials in Myotonic dystrophy in France (IV) prior to March 2025

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