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rAAVrh 74 MCK GALGT2

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Drug Profile

rAAVrh 74 MCK GALGT2

Alternative Names: Galgt2 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics; GalNAc tranferase gene therapies; rAAVrh74.MCK.GALGT2

Latest Information Update: 21 Oct 2020

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Nationwide Children's Hospital; Sarepta Therapeutics
  • 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 21 Oct 2020 Phase I/II development is still ongoing for Duchenne muscular dystrophy in USA
  • 20 Feb 2020 Nationwide Children's Hospital completes enrolment in a phase I/IIa trial in Duchenne muscular dystrophy (In children, In adolescents, In adults) in USA (Intra-arterial) (NCT03333590)
  • 06 Nov 2017 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children, In adolescents, In adults) in USA (Intra-arterial) (NCT03333590)

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