MYO 101

Drug Profile

MYO 101

Alternative Names: scAAVrh74.tMCK.hSGCB

Latest Information Update: 22 Dec 2017

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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
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Limb girdle muscular dystrophies

Most Recent Events

  • 20 Dec 2017 US FDA approves initiation of a phase I/IIa trial in Limb girdle muscular dystrophies (Myonexus Therapeutics pipeline, December 2017) before December 2017
  • 13 Dec 2017 Myonexus Therapeutics plans a phase I/IIa trial in Limb girdle muscular dystrophies (LGMD2E) in early 2018
  • 19 Oct 2017 Nationwide Children's Hospital has a published patent application with the Patent Coopertaion Treaty for adeno-associated virus vector delivery of β-sarcoglycan for treatment of Muscular dystrophy
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