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 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.

Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing therapeutics for patients with high unmet medical needs. The company's development candidates for Heme Malignancies includes P-BCMA-ALLO1, which is in Phase I trial to treat patients with relapsed/refractory multiple myeloma; P-CD19CD20-ALLO1, which is in Phase I trial for treating B cell malignancies and other autoimmune diseases; P-BCMACD19-ALLO1, an allogeneic, off-the-shelf CAR-T product candidate in preclinical development for treating multiple myeloma; and P-CD70-ALLO1 under preclinical development to treat hematological indications. It is also involved in the development of P-MUC1C-ALLO1 that is in Phase I trial for treating a range of solid tumors, including breast, colorectal, lung, ovarian, pancreatic, and renal cancers; P-PSMA-ALLO1, an autologous chimeric antigen receptor T cell (CAR-T) product candidate that is in preclinical development for the treatment of patients with metastatic castrate resistant prostate cancer (mCRPC); and P-PSMA-101, an allogeneic CAR-T product candidate under Phase 1 clinical trial for treating mCRPC. In addition, the company engages in the development of P-FVIII-101, a clinical stage liver-directed gene therapy for the in vivo treatment of hemophilia A; P-OTC-101, a clinical stage liver-directed gene therapy for the in vivo treatment of ornithine transcarbamylase deficiency; and P-PAH-101, a clinical stage liver-directed gene therapy for the in vivo treatment of phenylketonuria. It has a research collaboration and license agreement with F. Hoffmann-La Roche Ltd, and Hoffmann-La Roche Inc. The company was incorporated in 2014 and is headquartered in San Diego, California.

Allogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. It develops, manufactures, and commercializes UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate for the treatment of pediatric and adult patients with R/R CD19 positive B-cell acute lymphoblastic leukemia (ALL). The company also develops cemacabtagene ansegedleucel, an engineered allogeneic CAR T cell product candidate that targets CD19 for the treatment of large B-cell lymphoma; and is in Phase 1b clinical trial for the treatment of chronic lymphocytic leukemia. In addition, it is developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-605, an allogeneic CAR T cell product candidate that is in a Phase I clinical trial for the treatment of multiple myeloma; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; ALLO-316, an allogeneic CAR T cell product candidate that is in Phase 1 clinical trial for the treatment of advanced or metastatic RCC; ALLO-329 for the treatment of certain autoimmune diseases; DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors; and Claudin 18.2 for the treatment of gastric and pancreatic cancer. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc. It also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of allogeneic CAR T cell product candidates; and a strategic partnership with Foresight Diagnostics to develop MRD-based In-Vitro Diagnostic for use in ALPHA3. The company was incorporated in 2017 and is headquartered in South San Francisco, California.

Foghorn Therapeutics Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of medicines targeting genetically determined dependencies within the chromatin regulatory system in the United States. The company uses its proprietary Gene Traffic Control platform to identify, validate, and potentially drug targets within the system. It develops FHD-286, a small molecule inhibitor of the enzymatic activity of BRG1 and BRM that is in phase I for the treatment of relapsed and/or refractory acute myeloid leukemia/myelodysplastic syndrome. The company is also developing therapies for mutant cancers, such as Non-Small Cell Lung, bladder, endometrial, colorectal, and melanoma cancers; and dependent cancers, including prostate cancer and diffuse large b-cell lymphoma. It has a research collaboration and license agreement with Merck Sharp & Dohme Corp. to discover and develop novel oncology therapeutics against a transcription factor target; and Eli Lilly and Company for developing FHD-909, a selective ATPase inhibitor of BRM. Foghorn Therapeutics Inc. was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. It has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Caribou Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the development of genome-edited allogeneic cell therapies for the treatment of hematologic malignancies in the United States and internationally. Its lead product candidate is CB-010, an allogeneic anti-CD19 CAR-T cell therapy that is in phase 1 clinical trial to treat relapsed or refractory B cell non-Hodgkin lymphoma. The company also develops CB-011, an allogeneic anti-BCMA CAR-T cell therapy that is in phase 1 clinical trial for the treatment of relapsed or refractory multiple myeloma; and CB-012, an allogeneic anti-CD371 CAR-T cell therapy that is in phase 1 clinical trial for the treatment of relapsed or refractory acute myeloid leukemia. Caribou Biosciences, Inc. was incorporated in 2011 and is headquartered in Berkeley, California.

Vor Biopharma Inc. operates as a clinical-stage cell and genome engineering company. Its lead product is tremtelectogene empogeditemcel (trem-cel), an engineered hematopoietic stem cell (eHSC) product candidate that is in phase 1/2 trial to treat acute myeloid leukemia (AML) and other hematological malignancies. The company is also developing Mylotarg, a CD33-directed antibody drug conjugate (ADC) therapy for patients with leukemia; VCAR33, a CAR-T therapy designed to target CD33, a clinically validated target for AML, which are under preclinical and Phase 1/2 clinical studies; trem-cel + VCAR33 treatment system that is under preclinical studies for the treatment of AML; and CD33-CLL1 treatment system made with CD33-CLL1 multiplex-engineered eHSC and CD33-CLL1 multi-specific CAR-T, which are under preclinical studies for the treatment of AML. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.