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.

Precigen, Inc. operates as a discovery and clinical-stage biopharmaceutical company that develops gene and cell therapies using precision technology to target diseases in therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. It operates through two segments, Biopharmaceuticals and Exemplar. The company offers therapeutic platforms consisting of UltraCAR-T to provide chimeric antigen receptor T cell therapies for cancer patients; AdenoVerse immunotherapy, which utilizes a library of proprietary adenovectors for gene delivery of therapeutic effectors, immunomodulators, and vaccine antigen; and ActoBiotics for specific disease modification. It also develops programs based on the UltraCAR-T platform, including PRGN-3005 in Phase 1b clinical trial to treat advanced ovarian, fallopian tube, or primary peritoneal cancer; PRGN-3006 in Phase 1b trial for patients with relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndromes; and PRGN-3007 in Phase 1/1b trial for the treatment of advanced receptor tyrosine kinase-like orphan receptor 1-positive, hematologic, and solid tumors. In addition, the company is developing programs based on the AdenoVerse immunotherapy platform comprising PRGN-2009 in Phase 2 trial for patients with HPV-associated cancer; and PRGN-2012 in Phase ½ trial to treat recurrent respiratory papillomatosis, as well as AG019, which is based on the ActoBiotics platform and in Phase 1b/2a trial, to treat type 1 diabetes mellitus. Further, it provides UltraPorator, a proprietary electroporation device; and develops research models and services for healthcare research applications. The company was formerly known as Intrexon Corporation and changed its name to Precigen, Inc. in February 2020. Precigen, Inc. was founded in 1998 and is headquartered in Germantown, Maryland.

Editas Medicine, Inc., a clinical stage genome editing company, focuses on developing transformative genomic medicines to treat a range of serious diseases. It develops a proprietary gene editing platform based on CRISPR technology. The company develops EDIT-101, which is in Phase 1/2 BRILLIANCE trial for Leber Congenital Amaurosis; and reni-cel, a clinical development gene-edited medicine to treat sickle cell disease and transfusion-dependent beta-thalassemia. In addition, the company is developing alpha-beta T cells for solid and liquid tumors; and gamma delta T cell therapies to treat cancer. It has a research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; collaboration and nonexclusive license agreement with Genevant Sciences to develop Novel mRNA-LNP gene editing therapeuticsand strategic alliance and option agreement with Allergan Pharmaceuticals International Limited, as well as collaboration with Bristol Myers Squibb for research and development of alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was incorporated in 2013 and is based in Cambridge, Massachusetts.

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.

Precision BioSciences, Inc., an advanced gene editing company, develops in vivo gene editing therapies for gene edits, including gene elimination, insertion, and excision in the United States. The company offers ARCUS, a genome editing platform to DNA genome insertion, deletion, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; PBGENE-PMM for the treatment of m.3243 associated primary mitochondrial myopathy (PMM) which is expected to submit an IND and/or CTA. In addition, it develops PBGENE-NVS for sickle cell disease/beta thalassemia for insertion; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LL2 (insertion), a liver directed target; PBGENE-LL3, a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. The company has license and collaboration agreement with Caribou Biosciences, Inc.; license agreement with TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; development and license agreement with Eli Lilly and Company for the research and development of potential in vivo therapies for genetic disorders; Cellectis S.A.; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; Duke University; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.