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CRD TMH 001

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Drug Profile

CRD TMH 001

Alternative Names: CRD-TMH-001

Latest Information Update: 26 Sep 2025

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At a glance

  • Originator Cure Rare Disease; University of Massachusetts System
  • Developer Cure Rare Disease
  • Class Gene therapies
  • Mechanism of Action Dystrophin expression modulators; Gene modulators
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • No development reported Duchenne muscular dystrophy

Most Recent Events

  • 26 Sep 2025 No development reported - Phase-I for Duchenne muscular dystrophy (In adults) in USA (IV)
  • 31 Aug 2022 Phase-I clinical trials in Duchenne muscular dystrophy (In adults) in USA (IV) (NCT05514249)

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