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Drug Profile


Alternative Names: AAV1 vectored follistatin gene therapy - Milo Biotechnology; AAV1-Follistatin; Gene therapy-delivered myostatin inhibitor (AAV1-FS344) – Milo Biotechnology; rAAV1 CMV huFollistatin 344; rAAV1-Follistatin; rAAV1.CMV.huFollistin344

Latest Information Update: 28 Jan 2020

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Milo Biotechnology; Nationwide Children's Hospital
  • Class Anti-inflammatories; Gene therapies
  • Mechanism of Action Follistatin stimulants; Gene transference; Myostatin inhibitors; TGF-beta superfamily protein inhibitors
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy; Inclusion body myositis
  • New Molecular Entity No

Highest Development Phases

  • No development reported Duchenne muscular dystrophy; Inclusion body myositis

Most Recent Events

  • 28 Jan 2020 No development reported - Phase-I/II for Duchenne muscular dystrophy (In adolescents, In children, In the elderly, In adults) in USA (IM)
  • 28 Jan 2020 No development reported - Phase-I/II for Duchenne muscular dystrophy (In adults) in USA (IM)
  • 28 Jan 2020 No development reported - Phase-I/II for Inclusion body myositis (In adults) in USA (IM)
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