ACE-031, also referred to as ACVR2B or ActRIIB-IgG1, is a synthetically produced protein that was originally developed by two bio-pharmaceutical companies, namely Acceleron and Shire, for its potential ability to inhibit myostatin and other naturally occurring proteins that limit muscle growth. It was hypothesized that ACE-031 would act as a decoy receptor for the catabolic proteins, specifically myostatin, and bind to them in an effort to keep them away from muscle fibers. Numerous studies have been conducted to determine if ACE-031 is an effective treatment for children who have Duchenne Muscular Dystrophy (DMD).

 

What is Myostatin?

Myostatin, abbreviated GDF-8 (Growth Differentiation Factor-8), is a protein naturally produced in mammals, which acts on muscle cells as an inhibitor of myogenesis (differentiation and growth of muscle cells). The gene encoding myostatin was discovered in 1997 by geneticists Se-Jin Lee and Alexandra McPherron who produced a strain of mutant mice that lack the gene. These scientists produced a strain of mice that lacked the gene and had approximately twice as much muscle as normal mice.