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Agios Pharmaceuticals Inc is a biopharmaceutical company focused on the field of cellular metabolism to create differentiated medicines for rare diseases, with a focus on classical hematology. The company's primary focus is to develop potentially transformative small-molecule medicines. Its product candidate, Pyrukynd (mitapivat), is an activator of both wild-type and mutant pyruvate kinase enzymes, developed for the treatment of hemolytic anemias. The other drug candidates in its pipeline include Tebapivat (PK activator), being developed as a potential treatment for MDS-associated anemia and sickle cell disease, AG-181 (PAH stabilizer), AG-236, and others.
Edgewise Therapeutics Inc is a clinical-stage biopharmaceutical company. It is focused on the discovery, development, and commercialization of treatments for severe, rare muscle disorders. It is developing orally bioavailable, small molecule therapies for musculoskeletal diseases. The company's product candidates are Sevasemten and EDG-7500. Sevasemten is an orally administered small molecule designed to address the root cause of dystrophinopathies including Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker). The company is currently studying Sevasemten in Phase 2 trials, which are being held in the U.S. and Israel and in certain countries in Europe and Australasia.