Adverum Biotechnologies
NASDAQ:ADVMAdverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.
Revolution Medicines
NASDAQ:RVMDRevolution Medicines, Inc., a clinical-stage precision oncology company, develops novel targeted therapies for RAS-addicted cancers. The company's research and development pipeline comprises RAS(ON) inhibitors designed to be used as monotherapy in combination with other RAS(ON) inhibitors and/or in combination with RAS companion inhibitors or other therapeutic agents, and RAS companion inhibitors for combination treatment strategies. Its RAS(ON) inhibitors include RMC-6236 (multi), RMC-6291 (G12C), and RMC-9805 (G12D), which are in phase 1 clinical trial; and development candidates comprise RMC-5127 (G12V), RMC-0708 (Q61H), and RMC-8839 (G13C), as well as programs focused on G12R and other targets. The company's RAS companion inhibitors include RMC-4630 that is in phase 2 clinical trial; and RMC-5552, which is in phase 1 clinical trial. Revolution Medicines, Inc. was incorporated in 2014 and is headquartered in Redwood City, California.
Sangamo Therapeutics
NASDAQ:SGMOSangamo Therapeutics, Inc., a clinical-stage genomic medicine company, focuses on translating science into medicines that transform the lives of patients and families afflicted with serious diseases in the United States. The company's clinical-stage product candidates are ST-920, a gene therapy product candidate, which is in Phase 1/2 clinical study for the treatment of Fabry disease; TX200, a chimeric antigen receptor engineered regulatory T cell (CAR-Treg) therapy product candidate that is in Phase 1/2 clinical study for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation; SB-525, a gene therapy product candidate, which is in Phase 3 clinical trial for the treatment of moderately severe to severe hemophilia A; BIVV003, a zinc finger nuclease gene-edited cell therapy product candidate that is in Phase 1/2 PRECIZN-1 clinical study for the treatment of sickle cell disease. Its preclinical development products focus on CAR-Treg cell therapies for autoimmune disorders and genome engineering for neurological diseases. Sangamo Therapeutics, Inc. has collaborative and strategic partnerships with Biogen MA, Inc.; Kite Pharma, Inc.; Pfizer Inc.; Sanofi S.A.; Novartis Institutes for BioMedical Research, Inc.; Shire International GmbH; Dow AgroSciences LLC; Sigma-Aldrich Corporation; Genentech, Inc.; Open Monoclonal Technology, Inc.; F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; and California Institute for Regenerative Medicine. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was incorporated in 1995 and is headquartered in Brisbane, California.
SpringWorks Therapeutics
NASDAQ:SWTXSpringWorks Therapeutics, Inc., a commercial-stage biopharmaceutical company, engages in the development and commercialization of medicines for underserved patient populations suffering from rare diseases and cancer. Its lead product candidate is OGSIVEO (nirogacestat), an oral small molecule gamma secretase inhibitor that is in Phase III DeFi trial for the treatment of desmoid tumors; and Nirogacestat, is also in Phase 2 clinical development as a monotherapy for the treatment of ovarian granulosa cell tumors (GCT), a subtype of ovarian cancer. The company is also involved in the development of mirdametinib, an oral small molecule MEK inhibitor that is in Phase 2b clinical trials for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN); mirdametinib + lifirafenib, a combination therapy that is in Phase 1b clinical trial in patients with advanced or refractory solid tumors; and mirdametinib in monotherapy and combination approaches to treat other genetically defined solid tumors, including Phase 1/2 clinical trial for the treatment of pediatric and young adult patients with low-grade gliomas. In addition, it develops Brimarafenib (BGB-3245), an oral selective small molecule inhibitor of monomeric and dimeric forms of activating BRAF mutations. The company has collaborations with BeiGene, Ltd. and GlaxoSmithKline LLC; and license agreements with Pfizer Inc. for nirogacestat and mirdametinib. It also has a license agreement with Katholieke Universiteit Leuven and the Flanders Institute for Biotechnology for a portfolio of novel small molecule inhibitors of the TEA Domain; and Dana-Farber Cancer Institute for a portfolio of novel small molecule inhibitors of Epidermal Growth Factor Receptor. The company was founded in 2017 and is headquartered in Stamford, Connecticut.
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.