AAV1-FS344Alternative 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
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.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity No
Highest Development Phases
- Phase I/II Duchenne muscular dystrophy; Inclusion body myositis