Beam Therapeutics Inc. Common Stock
Beam 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; BEAM-302, a liver-targeting LNP formulation to treat severe alpha-1 antitrypsin deficiency; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease 1a. It also intends to develop BEAM-103, an anti-CD117 monoclonal antibody, and BEAM-104, a cell therapy for development in sickle cell disease and beta-thalassemia; and BEAM-201 and other potential ex vivo CAR-T therapies. 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.
Open BEAM dashboard ↗Solid Biosciences Inc. Common Stock
Solid Biosciences Inc. develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, which is in phase 1/2 for the treatment of Duchenne muscular dystrophy. It also develops SGT-212 for the treatment of Friedreich's ataxia that is in phase 1b clinical trial; SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia that is in preclinical phase; SGT-601 the treatment of TNNT2-mediated dilated cardiomyopathy, which is in preclinical phase; SGT-401 for the treatment of BAG3-mediated dilated cardiomyopathy that is in preclinical phase; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, which is in discovery phase; and other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries; genetic regulators, such as promoters, UTRs, and introns; immunomodulation technologies; manufacturing purity; and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc.; and a license agreement with the University of Washington, University of Missouri, University of Florida, and ICS Maugeri S.p.A. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.
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