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FB-606
- Development stage
- Preclinical
- Lead developer
- Fortress Biotech
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intramuscular
01
Overview
FB-606 is a preclinical-stage gene therapy candidate developed by Fortress Biotech (formerly Coronado Biosciences) for the treatment of Duchenne Muscular Dystrophy (DMD). The therapy utilizes an adeno-associated virus (AAV) vector to deliver the gene for follistatin, a naturally occurring protein that acts as a potent antagonist to myostatin (growth differentiation factor 8). Myostatin is a key negative regulator of muscle mass; by overexpressing follistatin, FB-606 aims to inhibit myostatin signaling, thereby promoting muscle hypertrophy and potentially slowing the progressive muscle degeneration and weakness characteristic of DMD.
- Other names
- AAV-follistatinrAAV1-CMV-huFollistatin344rAAV-1-CMV-huFollistatin344rAAV 1-CMV-huFollistatin344
02
Targets
FST (Follistatin)GDF8 (Myostatin)
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