Apitegromab (SRK-015) is a fully human
monoclonal antibody
A monoclonal antibody (mAb, more rarely called moAb) is an antibody produced from a cell lineage made by cloning a unique white blood cell. All subsequent antibodies derived this way trace back to a unique parent cell.
Monoclonal antibodie ...
developed to treat
spinal muscular atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood and if left untreated it is the most common geneti ...
. It works by binding to and inhibiting
promyostatin, a precursor to
myostatin
Myostatin (also known as growth differentiation factor 8, abbreviated GDF8) is a protein that in humans is encoded by the ''MSTN'' gene. Myostatin is a myokine that is produced and released by myocytes and acts on muscle cells to inhibit muscle ...
, which limits the size of skeletal muscle tissue, as well as inactive myostatin. It does not bind to active myostatin,
activin A
Activin and inhibin are two closely related protein complexes that have almost directly opposite biological effects. Identified in 1986, activin enhances FSH biosynthesis and secretion, and participates in the regulation of the menstrua ...
, active
BMP9/10 or
TGFβ1 that all operate on the
activin type 2 receptors.
References
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Monoclonal antibodies
Spinal muscular atrophy
Myostatin inhibitors