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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.
Mesoblast Ltd is a commercial-stage biotechnology company and a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The company's product candidates include: Ryoncil (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease, as well as for the treatment of moderate to severe acute respiratory distress syndrome; Revascor (rexlemestrocel-L) for the treatment of chronic heart failure; and Rexlemestrocel-L for chronic low back pain. The company is managed as one operating segment, being the development of an adult stem cell technology platform for commercialization.