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WAVE Life Sciences Ltd is unlocking the broad potential of RNA medicines also known as oligonucleotides, it targeting ribonucleic acid (RNA), to transform human health. RNA medicines platform, PRISMTM, combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. Its toolkit of RNA-targeting modalities includes RNA editing, splicing, antisense silencing, and RNA interference, providing capabilities for designing and sustainably delivering candidates that optimally address disease biology. Its programs are for rare and prevalent diseases, including alpha-1 antitrypsin deficiency, obesity, Duchenne muscular dystrophy, and Huntington's disease.
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.