Companies in #CNS

Develops and commercializes medicines for neurological disorders and rare diseases, including Parkinson's disease psychosis, Alzheimer's disease psychosis, and Rett syndrome.

Alnylam Pharmaceuticals (NASDAQ: ALNY) is the leading RNAi therapeutics company, with six approved products (Onpattro, Givlaari, Oxlumo, Amvuttra, Qfitlia, and the Leqvio collaboration with Novartis) and a deep pipeline in cardiometabolic, hepatic infectious disease, and CNS indications. Alnylam's approved medicines target transthyretin amyloidosis (Onpattro, Amvuttra), acute hepatic porphyria (Givlaari), primary hyperoxaluria type 1 (Oxlumo), and hemophilia A and B (Qfitlia). Leqvio (inclisiran), licensed to and commercialized by Novartis, targets LDL cholesterol.

Clinical-stage biopharmaceutical company developing novel small-molecule therapeutics for rare genetic neurodevelopmental, neuromuscular and central nervous system disorders.

Hansoh Pharmaceutical Group is a China-based biopharmaceutical company with marketed products and development programs across oncology, central nervous system disorders, anti-infectives, metabolic disease, and autoimmune disease.

Indivior PLC (NASDAQ: INDV) is a specialty pharmaceutical company singularly focused on opioid use disorder (OUD), developing and commercializing evidence-based treatments such as the once-monthly injection Sublocade to support long-term recovery.

Jupiter Neurosciences is a clinical-stage pharmaceutical company developing treatments for central nervous system (CNS) disorders, with a focus on psychedelic-assisted therapy and neurodegenerative diseases. The company is advancing JNS-101 (MDMA analog) and other CNS-targeting candidates.

UK-based drug discovery and development company focused on selective mitophagy modulation through USP30 inhibition for CNS and kidney disease.

Mitsubishi Tanabe Pharma is a Japanese pharmaceutical company with a portfolio spanning immunology, central nervous system disorders, diabetes, and vaccines. It is the originator of Radicava (edaravone) for ALS and has been divesting non-core plants as part of a post-buyout strategic pivot.

PureTech Health is a clinical-stage biotherapeutics company developing innovative medicines for serious diseases. It founded and spun out multiple companies including Karuna, Gelesis, and Celea, with a current focus on lymphatic and CNS-targeted therapeutics.

Washington, DC-based biopharmaceutical company developing and commercializing therapies for central nervous system disorders, dermatologic conditions, and rare diseases, with three FDA-approved products on market.