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 ↗Lexeo Therapeutics, Inc. Common Stock
Lexeo Therapeutics, Inc., a clinical stage genetic medicine company, focuses on hereditary and acquired diseases with high unmet need in the United States. Its product pipeline comprises LX2006, an AAVrh10-based gene therapy candidate, which is in phase 1/2 clinical trial to treat friedreich ataxia cardiomyopathy; LX2020, an AAVrh10-based gene therapy candidate, which is in phase 1/2 to deliver a functional plakophilin-2 (PKP2) gene to cardiac muscle for the treatment of PKP2-ACM; and LX2021, a gene therapy candidate, which is in preclinical trial to deliver the coding sequence for the functional connexin 43, or Cx43, protein for a group of inherited cardiac muscle disorders associated with a high risk of sudden death, including arrhythmogenic cardiomyopathy (ACM) and certain forms of dilated cardiomyopathy. The company also develops LX2022, a gene therapy candidate, which is in preclinical trial to deliver a functional TNNI3 gene to myocardial cells to treat a distinct form of hypertrophic cardiomyopathy. In addition, it is developing LX1001, an AAVrh10-based gene therapy candidate, which has completed phase 1/2 clinical trial for the treatment of APOE4-associated Alzheimer's disease. The company has license agreement with Cornell University to conduct of the Phase 1/2 clinical trial of LX1001, as well as support the development of LX1004 program; research collaboration agreement with Cornell University to conduct preclinical research to develop the licensed technology; and third license agreement which obtained certain rights for FA cardiomyopathy, including rights to current and future clinical data from an ongoing Cornell University investigator-initiated Phase 1A trial of a gene therapy candidate. The company was founded in 2017 and is headquartered in New York, New York.
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