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.CMV.huFollistin344

Latest Information Update: 12 Oct 2017

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Milo Biotechnology; Nationwide Children's Hospital
  • Class 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

  • Phase I/II Duchenne muscular dystrophy; Inclusion body myositis

Most Recent Events

  • 12 Oct 2017 Phase-I/II development in Becker muscular dystrophy and Duchenne muscular dystrophy is ongoing in USA (IM) (Milo Biotechnology pipeline, October 2017)
  • 08 Sep 2017 Phase I/II development in Inclusion body myositis is ongoing in USA (IM)
  • 28 Mar 2017 Nationwide Children's Hospital completes enrolment in its phase I/II trial for Duchenne Muscular Dystrophy (In children, In adolescents, In adults) in USA (NCT02354781)
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