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.
Fate Therapeutics
NASDAQ:FATEFate Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops programmed cellular immunotherapies for cancer and immune disorders worldwide. The company's chimeric antigen receptor (CAR)-targeted NK and T-cell product candidates include FT576 to treat multiple myeloma, and FT522, to treat lymphoma and autoimmune disorders. Its CAR T-cell programs include FT819 to treat hematologic malignancies and solid tumors, and FT825 to treat solid tumors. The company has a collaboration and option agreement with Ono Pharmaceutical Co. Ltd. for the development and commercialization of off-the-shelf, iPSC-derived CAR T-cell product candidates for the treatment of cancer. Fate Therapeutics, Inc. was incorporated in 2007 and is headquartered in San Diego, California.
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.
Twist Bioscience
NASDAQ:TWSTTwist Bioscience Corporation engages in the manufacture and sale of synthetic DNA-based products. The company offers synthetic genes and gene fragments used in product development for therapeutics, diagnostics, chemicals/materials, food/agriculture, data storage, and various applications within academic research by biotech, pharma, industrial chemical, and agricultural companies, as well as academic labs; Oligo pools used in targeted NGS, CRISPR gene editing, mutagenesis experiments, DNA origami, DNA computing, data storage in DNA, and other applications; and immunoglobulin G proteins for customers focused on the pursuit of drug discovery and development. It also provides NGS tools comprising library preparation kits, human exome kits, and fixed and custom panels, as well as alliance panels, primarily used within diagnostic tests for various indications, population genetics research and biomarker discovery, translational research, microbiology, and applied markets research; and methylation detection kits for cancer, and rare and inherited disease study, as well as fast hybridization and full RNA sequencing workflow solution. In addition, the company provides synthetic SARS-CoV-2 RNA reference sequences as positive controls for the development of both NGS and reverse transcription-polymerase chain reaction assays; synthetic monkeypox controls; and various respiratory viral controls, including for influenzas, respiratory syncytial virus, rhinoviruses, SARS, MERS, and coronaviruses. Further, it offers SARS-CoV-2 Research Panels, such as the Twist Respiratory Virus Panel and the Pan-Viral Research Panel, for the detection of disease in a research setting; precision DNA libraries used for antibody engineering, affinity maturation, and humanization; and antibody optimization solution to enable simultaneous optimization of multiple characteristics of a given antibody. The company was incorporated in 2013 and is headquartered in South 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.