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Sarepta Therapeutics Inc is a biotechnology company focused on treating rare, infectious, and other diseases. It targets a broad range of diseases while focusing on the rapid development of its drug candidates. Sarepta's involves proprietary RNA-targeted technology platforms to be used for developing novel pharmaceutical products to treat a broad range of diseases and address key unmet medical needs. The company uses third-party contractors to manufacture its product candidates. Majority of company's product candidates are at an early stage of development.
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.