Myostatin or growth differentiation factor 8 (GDF8) is a naturally occurring protein in muscles. It belongs to a family of proteins called transforming growth factor-beta or TGFβ. Myostatin, produced through instructions from the MSTN gene, regulates muscle cell growth and differentiation. Specifically, it limits skeletal muscle growth to protect muscles from becoming excessively large.
Secreted by the muscle cells, myostatin production increases with age.
Myostatin inhibitors are a group of molecules that block myostatin, and may work to improve muscle mass and strength in children with muscle-wasting diseases. They are being investigated as potential treatments for diseases such as muscular dystrophy.
Myostatin inhibitors in development
Several myostatin inhibitors are being evaluated as potential treatments for different types of muscular dystrophy.
RG6206 (also known as RO7239361 or BMS986089) is an investigational molecule to possibly treat Duchene muscular dystrophy (DMD). It was first developed by Bristol-Myers Squibb and is now licensed to Roche. This molecule works by binding to myostatin and limiting its function, and so encourage muscle growth.
ACE-083 is an investigational therapy being developed by Acceleron to possibly treat facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease (CMT). It is a small molecule designed to bind to and block myostatin and another TGFβ protein called activin, increasing the strength and function of muscles.
Domagrozumab (PF-06252616) was a candidate intended to treat DMD being developed by Pfizer. A monoclonal antibody against myostatin, it was designed to work by binding to myostatin and blocking its activity. Development of domagrozumab was discontinued in August 2018 after it failed to show effectiveness against placebo in a Phase 2 study of ambulatory boys with DMD.
Last updates 07/15/2019
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