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 ↗Vor Biopharma Inc. Common Stock
Vor Biopharma Inc. operates as a clinical-stage cell and genome engineering company. The company offers the Vor Bio platform, a technology platform for the treatment of acute myeloid leukemia (AML) and other blood cancers. It develops tremtelectogene empogeditemcel (trem-cel), a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate to treat acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and other blood cancers; trem-cel in combination with Mylotarg to treat patients in VBP101, which is in phase 1 / 2; and VCAR33, a CAR-T therapy designed to target CD33, which is under preclinical studies to treat AML post-transplant. The company also develops trem-cel + VCAR33 treatment system, CD33-directed CAR-T program, which is under pre-clinical studies for the treatment of AML; VADC45 for the treatment of hematologic malignancies, conditioning, and immune reset, which is under preclinical studies; and CD33-CLL1 treatment system made with CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy, which is under preclinical studies for the treatment of AML. It has a license agreement with Columbia University and the National Institutes of Health for patents; and with Editas Medicine, Inc. for ex-vivo Cas9 gene-edited HSC therapies for the treatment and/or prevention of hematological malignancies. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
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