Beam Therapeutics
NASDAQ:BEAMBeam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. It develops BEAM-101 for the treatment of sickle cell disease or beta-thalassemia; and BEAM-302, a liver-targeting LNP formulation to treat severe alpha-1 antitrypsin deficiency; BEAM-201, an anti-CD7 CAR-T product candidate, which is in Phase 1/2 clinical trials for the treatment of refractory T-cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease 1a. The company has research collaboration agreement with Pfizer Inc., focus on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Apellis Pharmaceuticals to conduct preclinical research on target specific genes within the complement system in various organs, including the eye, liver, and brain; Verve Therapeutics, Inc., for cardiovascular disease treatments; Sana Biotechnology, Inc., to research, develop, and commercialize rights to CRISPR Cas12b for cell therapy programs; and Orbital Therapeutics design RNA for the prevention, treatment or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.
CRISPR Therapeutics
NASDAQ:CRSPCRISPR Therapeutics is a gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene-editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.
Neurocrine Biosciences
NASDAQ:NBIXNeurocrine Biosciences, Inc. discovers, develops, and markets pharmaceuticals for neurological, neuroendocrine, and neuropsychiatric disorders in the United States and internationally. The company's products include INGREZZA for tardive dyskinesia and chorea associated with Huntington's disease; ALKINDI for adrenal insufficiency; Efmody capsules for classic congenital adrenal hyperplasia; Orilissa tablets for endometriosis; and Oriahnn capsules to treat uterine fibroids. Its product candidates in clinical development include valbenazine to treat dyskinetic cerebral palsy in pediatrics and adults; NBI-921352 to treat developmental and epileptic encephalopathy syndrome in pediatrics and adults; NBI-827104 to treat epileptic encephalopathy with continuous spike-and-wave during sleep; NBI-1076986 to treat movement disorders; crinecerfront to treat congenital adrenal hyperplasia in adults and children; EFMODY to treat congenital adrenal hyperplasia and adrenal insufficiency in adults; valbenazine for the adjunctive treatment of schizophrenia; NBI-1065845 for the treatment of inadequate response to treatment in major depressive disorder; luvadaxistat to treat cognitive impairment related to schizophrenia; NBI-1117568 for the treatment of schizophrenia; NBI-1070770 to treat major depressive disorder; NBI-1117570 for the treatment of symptoms of psychosis and cognition in neurological and neuropsychiatric conditions; and NBI-1117569, NBI-1117567, and NBI-1065890 to treat CNS indications. The company also has license and collaboration agreements with Heptares Therapeutics Limited; Takeda Pharmaceutical Company Limited; Idorsia Pharmaceuticals Ltd; Xenon Pharmaceuticals Inc.; Voyager Therapeutics, Inc.; BIAL Portela & Ca, S.A.; Mitsubishi Tanabe Pharma Corporation; and AbbVie Inc. The company was incorporated in 1992 and is headquartered in San Diego, California.
Vir Biotechnology
NASDAQ:VIRVir Biotechnology, Inc., a commercial-stage immunology company, develops therapeutic products to treat and prevent serious infectious diseases. It offers tobevibart + elebsiran for the treatment of chronic hepatitis delta; elebsiran + PEG-IFN-a, tobevibart ± elebsiran ± PEG-IFN-a, and elebsiran+ TLR8+PD-1 for the treatment of chronic hepatitis B; VIR-1388 and Cure mAb combination for the treatment of human immunodeficiency virus; VIR-8190 for the treatment of respiratory syncytial virus / human metapneumovirus; VIR-2981 for the treatment of influenza; VIR-1949 for the treatment of pre-cancerous HPV lesions; and VIR07229 and Sotrovimab for the treatment of COVID-19 infection under the Xevudy brand. The company has grant agreements with Bill & Melinda Gates Foundation and National Institutes of Health; an option and license agreement with Brii Biosciences Limited; a collaboration and license agreement with Alnylam Pharmaceuticals, Inc.; license agreements with MedImmune; collaboration with WuXi Biologics and Glaxo Wellcome UK Ltd.; and a collaborative research agreement with GlaxoSmithKline Biologicals SA. It also has a manufacturing agreement with Samsung Biologics Co.,Ltd. for the manufacture of SARS-COV-2 antibodies. The company was incorporated in 2016 and is headquartered in San Francisco, California.
Acceleron Pharma
NASDAQ:XLRNAcceleron Pharma, Inc. is a biopharmaceutical company, which engages in the discovery, development and commercialization of therapeutics to treat serious and rare diseases. Its product candidates include Luspatercept, designed to patients with chronic anemia associated within a wide range of blood diseases; ACE-083, designed for the treatment of focal muscle disorders; and Sotatercept, designed to treat pulmonary arterial hypertension. The company was founded by John L. Knopf and Thomas P. Maniatis in June 2003 and is headquartered in Cambridge, MA.