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rAAV1.CMV.huFollistatin344
- Development stage
- Phase 2
- Lead developer
- Milo Biotechnology
- Modality
- Gene Therapies
- Administration
- Intramuscular
01
Overview
rAAV1.CMV.huFollistatin344 is an investigational gene therapy designed to deliver the human follistatin 344 gene using a recombinant adeno-associated virus serotype 1 (rAAV1) vector under the control of a cytomegalovirus (CMV) promoter. The expressed protein, follistatin 344, is a potent inhibitor of myostatin and other members of the TGF-beta superfamily, leading to increased muscle growth and reduced muscle degeneration. The primary mechanism involves antagonizing myostatin signaling in skeletal muscle by increasing local levels of follistatin, which binds and neutralizes myostatin and activin. This approach has been investigated for treating Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and inclusion body myositis. The drug was developed by Milo Biotechnology under license from Nationwide Children's Hospital[3][4][5][7].
- Other names
- Gene therapy-delivered myostatin inhibitor (AAV1-FS344)AAV1-FollistatinAAV-1-FollistatinAAV 1-Follistatin
02
Targets
Activin AINHBB (Activin B)GDF11 (Growth differentiation factor 11)GDF8 (Myostatin)
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