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A pharmaceutical company engaged in developing and commercializing central nervous system (CNS) therapies that address specific clinical needs for people living with orphan and other CNS disorders.
Dicerna Pharmaceuticals, Inc.
A ribonucleic acid (RNA) interference-based biopharmaceutical company, which silences genes and restores health.
A Danish biopharmaceutical company focused on the development of treatments for rare diseases in patients. The company has developed a drug against two rare eating disorders - hypothalamic obesity (HO) and Prader-Willi syndrome ( PWS).
Translate BIO, A Sanofi Company
A clinical-stage US biopharmaceutical company developing new generation messenger RNA (mRNA) based vaccines to save human lives.