Drug intelligence / Profile preview

follistatin

Development stage
Unknown
Lead developer
Minicircle
Modality
Recombinant Proteins and Enzymes, Gene Therapies
Administration
Intramuscular
01

Overview

Follistatin is a naturally occurring glycoprotein primarily produced in the liver and found throughout almost all body tissues. It functions as a potent antagonist of several members of the transforming growth factor-beta (TGF-β) superfamily, most notably activin and myostatin (GDF8). By binding to these proteins, especially myostatin, follistatin inhibits their activity, thereby promoting muscle growth and regeneration. This mechanism has made it a focus for research into therapies for muscle-wasting diseases such as muscular dystrophies, inclusion body myositis, spinal muscular atrophy, and other neuromuscular disorders. Several forms exist—follistatin 344 is commonly used in gene therapy approaches; follistatin 315 is the main circulating form targeting muscle tissue; and follistatin 288 plays roles in reproductive health and tumor biology. Experimental gene therapies using adeno-associated virus vectors to deliver the FS344 gene have shown increased muscle mass and strength in animal models and early human trials. Elevated levels of circulating follistatin are also associated with metabolic conditions like type 2 diabetes but may have both beneficial or detrimental effects depending on disease context[1][2][3][6][7][8][9].

Other names
follistatin 344follistatin344follistatin-344follistatin 315follistatin315follistatin-315follistatin 288follistatin288follistatin-288
02

Targets

GDF11 (Growth differentiation factor 11)GDF8 (Myostatin)Activin A

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