At Keros we seek to leverage our unique understanding of multiple pathways to develop therapeutics for treatment of rare muscle disorders.
Rare diseases are defined differently throughout the world, but in the United States they are considered to be any condition affecting less than 200,000 Americans at any time. Many rare diseases are genetic in origin and over 6000 different conditions have been identified. The vast majority of these have no effective treatment.
Neuromuscular diseases represent a wide range of disorders which can affect the body at any point from the peripheral nervous system, the neuromuscular junction or through to skeletal muscle, and may involve connective tissue. Although the onset, etiology, phenotype and mechanism by which these disorders are manifest can vary widely, the common symptoms are abnormal muscle function, resulting in muscle weakness and often loss of ambulation.
Many neuromuscular diseases, particularly in children, are genetic in origin. While the causes of such disorders are becoming better and better understood, treatments for many are still lacking. Additionally, many of the current standards of care address only the symptoms of the disease(s), rather than modifying the underlying cause of the disease.
Keros is currently exploring both small molecule and biologic approaches to treating some of the most intractable neuromuscular diseases. The Keros team has a deep understanding of the biology relevant to growth and development of skeletal muscle.
Keros’ lead program is focused on inhibition of ALK2, the genetic driver for the orphan disease fibrodysplasia ossificans progressive (FOP), which promotes the transformation of muscle into bone. ALK2 has been validated as a critical node through human genetics and in vivo data. The Keros small molecule ALK2 program, licensed from Partners and NCATS, is advancing to preclinical safety studies.
Keros has intellectual property covering a series of small molecule therapeutics currently in development for neuromuscular disorders and potentially other indications. We also have a number of biologics in development to improve muscle function, but which have shown efficacy in related conditions.