Drug Profile


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

Latest Information Update: 06 Oct 2016

$50 / €47 *
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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; Transforming growth factor beta 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

  • 04 Oct 2016 AAV1-FS344 receives Orphan Drug status for Inclusion body myositis in USA
  • 01 Jan 2015 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children, In adolescents, In adults) in USA (IM) (NCT02354781)
  • 03 Dec 2014 Milo Biotechnology has patent protection for AAV1-FS344 in USA
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