Follistatin gene therapy

Follistatin gene therapy is an investigational genetic treatment designed to increase muscle mass and strength by delivering the human follistatin (FST) gene to target tissues. Follistatin is a secreted glycoprotein that acts as a potent inhibitor of the myostatin pathway—a key negative regulator of skeletal muscle growth—by binding and neutralizing myostatin and other TGF-β family ligands such as activin. This results in increased muscle mass and improved cellular performance[1][2][5]. The most commonly used isoform for therapeutic purposes is FS344, which undergoes post-translational modification to become the serum-circulating FS315 variant with reduced affinity for activin compared to other isoforms[1][5]. Delivery methods include adeno-associated virus (AAV) vectors (e.g., AAV1-FS344), plasmid-based systems, or proteolipid vehicles[3][6][8]. The primary indications under investigation are neuromuscular disorders such as Becker muscular dystrophy and inclusion body myositis; additional research explores its potential in obesity prevention, metabolic disease mitigation, osteoarthritis management, age-related sarcopenia, and longevity enhancement[2][6][8]. Developers include Milo Biotechnology (AAV1-FS344), Oisín Biotechnologies (proteolipid vehicle delivery), Minicircle (plasmid-based system), among others[3][6][8].

Other names

Follistatin gene transferFST gene therapyFS344 gene therapyFS-344 gene therapyFS 344 gene therapy