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.
CymaBay Therapeutics
NASDAQ:CBAYCymaBay Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing and providing therapies to treat liver and other chronic diseases. Its lead product candidate is seladelpar (MBX-8025), a selective agonist of peroxisome proliferator activated receptor delta for the treatments of primary biliary cholangitis (PBC). The company also develops MBX-2982, a G protein-coupled receptor 119 (GPR119) agonist, in subjects with type 1 diabetes. It has a license agreement with ABW Cyclops SPV LP to support development of seladelpar for the treatment of PBC. The company was formerly known as Metabolex, Inc. CymaBay Therapeutics, Inc. was incorporated in 1988 and is headquartered in Fremont, California.
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.
Gilead Sciences
NASDAQ:GILDGilead Sciences, Inc., a biopharmaceutical company, discovers, develops, and commercializes medicines in the areas of unmet medical need in the United States, Europe, and internationally. The company provides Biktarvy, Genvoya, Descovy, Odefsey, Truvada, Complera/ Eviplera, Stribild, Sunlencs, and Atripla products for the treatment of HIV/AIDS; Veklury, an injection for intravenous use, for the treatment of COVID-19; and Epclusa, Harvoni, Vemlidy, and Viread for the treatment of viral hepatitis. It also offers Yescarta, Tecartus, and Trodelvy products for the treatment of oncology; Letairis, an oral formulation for the treatment of pulmonary arterial hypertension; and AmBisome, a liposomal formulation for the treatment of serious invasive fungal infections. The company has collaboration agreements with Arcus Biosciences, Inc.; Merck Sharp & Dohme Corp.; Pionyr Immunotherapeutics Inc.; Tizona Therapeutics, Inc.; Galapagos NV; Janssen Sciences Ireland Unlimited Company; Japan Tobacco, Inc.; Dragonfly Therapeutics, Inc.; Arcellx, Inc.; Everest Medicines; Merck & Co, Inc.; Tentarix Biotherapeutics Inc.; and Assembly Biosciences, Inc. It also has research collaboration, option, and license agreement with Merus N.V. for the discovery of novel dual tumor-associated antigens (TAA) targeting trispecific antibodies. The company was incorporated in 1987 and is headquartered in Foster City, California.
ImmunityBio
NASDAQ:IBRXImmunityBio, Inc., a clinical-stage biotechnology company, engages in discovering, developing, and commercializing next-generation immuno- and cellular therapies for cancer and infectious diseases in the United States and Europe. The company offers immunotherapy and cell therapy platforms, including antibody cytokine fusion protein N-803, vaccine delivery technologies, and natural killer cell therapies. Its lead biologic commercial product candidate is Anktiva, an IL-15 superagonist antibody-cytokine fusion protein for the treatment of bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without Ta or T1 disease. The company also develops other therapeutic agents for the treatment of liquid and solid tumors, including bladder, lung, colorectal, and ovarian cancers, as well as glioblastoma multiforme, acute myeloid leukemia, and human immunodeficiency virus. It has collaboration agreements with National Cancer Institute and Amyris, Inc.; and license agreements with LadRx Corporation, GlobeImmune, Inc., Access to Advanced Health Institute, 3M Innovative Properties Company, Sanford Health, Shenzhen Beike Biotechnology Co. Ltd., and Viracta Therapeutics, Inc. The company is based in San Diego, California.
Madrigal Pharmaceuticals
NASDAQ:MDGLMadrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development of therapeutics for the treatment of non-alcoholic steatohepatitis (NASH) in the United States. Its lead product candidate is resmetirom, a liver-directed thyroid hormone receptor beta agonist, which is in Phase 3 clinical trials for treating NASH. The company is headquartered in West Conshohocken, Pennsylvania.
Intellia Therapeutics
NASDAQ:NTLAIntellia Therapeutics, Inc., a genome editing company, focuses on the development of curative therapeutics. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; NTLA-2002 for the treatment of hereditary angioedema; and NTLA-3001 for alpha-1 antitrypsin deficiency associated lung disease. It also focusses on programs comprising hemophilia A and hemophilia B; and research of proprietary programs focused on developing engineered cell therapies to treat various cancers and autoimmune diseases. In addition, the company offers tools comprising of Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. It has license and collaboration agreement with Regeneron Pharmaceuticals, Inc. to co-develop potential products for the treatment of hemophilia A and hemophilia B; AvenCell Therapeutics, Inc. to develop allogeneic universal CAR-T cell therapies, and co-develop and co-commercialize allogeneic universal CAR-T cell products for an immuno-oncology indication; SparingVision SAS to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases; Kyverna Therapeutics, Inc. for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases; and ONK Therapeutics, Ltd. for the development of engineered NK cell therapies to cure patients with cancer. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.