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MYO 103

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Drug Profile

MYO 103

Alternative Names: MYO103; rAAVrh74.MHCK7.hSGCG; SRP-9005

Latest Information Update: 23 Apr 2025

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Myonexus Therapeutics; Nationwide Children's Hospital
  • Class Gene therapies
  • Mechanism of Action Gene transference; Sarcoglycan replacements
  • 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

  • Preclinical Limb girdle muscular dystrophies

Most Recent Events

  • 01 May 2025 Sarepta Therapeutics plans a phase III COMPASS trial for Limb girdle muscular dystrophies (In children, In adults, In adolescents, In the elderly) in USA (IV, infusion) (NCT06952686)
  • 23 Apr 2025 MYO 103 is still in preclinical development in Limb-girdle-muscular-dystrophies in USA (Parenteral)
  • 15 Apr 2025 US FDA clears Sarepta Therapeutics to proceed with dosing in clinical trial (COMPASS) for Limb girdle muscular dystrophies in USA (Parenteral)

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