CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. Its CRISPR/Cas9 is a gene editing technology that allows for precise directed changes to genomic DNA. The company has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, immune-oncology and autoimmune, in vivo, and type 1 diabetes. The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease, and hemoglobinopathies in which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting CD19 and CTX131 targeting CD70 for oncology and autoimmunune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and VCTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.

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.

Intellia 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.

Verve Therapeutics, Inc., a clinical stage genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases in the United States. The company's lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver; and VERVE-102, a product candidate that targets the PCSK9 gene for the treatment of HeFH. It engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.

Prime Medicine, Inc., a biotechnology company, delivers genetic therapies to address the spectrum of diseases by deploying gene editing technology. The company offers Prime Editors with a Prime Editor protein, comprising a fusion between a Cas protein and a reverse transcriptase enzyme; and a pegRNA, which targets the Prime Editor to a specific genomic location and provides a template for making the desired edit to the target DNA sequence. It has a research collaboration with Cimeio Therapeutics to develop Prime Edited Shielded-Cell & Immunotherapy Pairs for genetic diseases, acute myeloid leukemia, and myelodysplastic syndrome. The company has a strategic research collaboration and license agreement with bristol myers squibb to develop and commercialize multiple prime edited ex vivo t-cell therapies. Prime Medicine, Inc. was incorporated in 2019 and is based in Cambridge, Massachusetts.

Cellectis S.A., a clinical stage biotechnological company, develops products based on gene-editing with a portfolio of allogeneic chimeric antigen receptor T-cells product candidates. The company is developing ALLO-715 and ALLO-605 for the treatment of multiple myeloma; ALLO-501 to treat large B-cell lymphoma; ALLO-316, an allogeneic engineered CAR T-cell product targeting CD70; ALLO-819 to treat acute myeloid leukemia; ALLO-213 for the treatment of small cell lung cancer; and ALLO-182 to treat gastric and pancreatic cancer. It also develops UCART22 for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia; UCART123 to treat acute myeloid leukemia; UCART 20x22 for relapsed or refractory B-Cell non-hodgkin's lymphoma; and cema-cel to treat relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma. The company has strategic alliances with Allogene Therapeutics, Inc. and Les Laboratoires Servier; research collaboration and exclusive license agreement with Iovance Biotherapeutics, Inc.; and joint research and collaboration, and investment agreement with AstraZeneca Holdings B.V., as well as with AstraZeneca Ireland Limited. Cellectis S.A. was founded in 1999 and is headquartered in Paris, France.

Metagenomi, Inc., a genetic medicines company, develops therapeutics for patients using metagenomics-derived genome editing toolbox in the United States. The company's genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems, such as prime editing systems and clustered regularly interspaced short palindromic repeat-associated transposases. It has a development, option, and license agreement with Affini-T Therapeutics, Inc. to develop and commercialize gene edited T-cell receptor-based therapeutic products in the field of treatment, prevention, or diagnosis of human cancer; and a collaboration and license agreement with Ionis Pharmaceuticals, Inc. to research, develop, and commercialize investigational medicines using genome editing technologies. The company was incorporated in 2016 and is based in Emeryville, California.

Generation Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.