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.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy. It also develops RGX-121 for the treatment of mucopolysaccharidosis type II that is in Phase III clinical trial; RGX-111 for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II; and RGX-381 to treat the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.

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.

Genelux Corporation, a clinical-stage biopharmaceutical company, focuses on developing next-generation oncolytic viral immunotherapies for patients suffering from aggressive and/or difficult-to-treat solid tumor types. Its lead product candidate is Olvi-Vec, a proprietary modified strain of the vaccinia virus for the treatment of ovarian cancer and non-small cell lung cancer. The company is also developing V2ACT Immunotherapy for treating pancreatic cancer. Genelux Corporation has a licensing agreement with ELIAS Animal Health, LLC for V-VET1, a clinical stage animal health product candidate. The company was incorporated in 2001 and is headquartered in Westlake Village, 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.

RenovoRx, Inc., a clinical-stage biopharmaceutical company, focuses on developing proprietary targeted combination therapies to improve therapeutic outcomes for cancer patients undergoing treatment. Its lead product candidate is RenovoGem, an oncology drug-device combination product, consisting of intra-arterial gemcitabine and RenovoCath that is in Phase III clinical trials for the locally advanced pancreatic cancer. The company has a research collaboration with Imugene Limited to deliver oncolytic virus therapy for the treatment of difficult-to-access tumors. RenovoRx, Inc. was founded in 2009 and is headquartered in Los Altos, California.

Alaunos Therapeutics, Inc., a clinical-stage oncology-focused cell therapy company, develops adoptive T-cell receptor (TCR) engineered T-cell therapies (TCR-T) to treat multiple solid tumor types. It develops Library TCR-T Cell Theraphy, which is in Phase I/II clinical trial for 12 TCRs reactive to mutated KRAS, TP53, and EGFR from its TCR library for the treatment of non-small cell lung, colorectal, endometrial, pancreatic, ovarian, and bile duct cancers. The company is also developing hunTR, a human neoantigen TCR discovery engine; and Sleeping Beauty Gene Transfer Platform, a non-viral genetic engineering technology. The company was founded in 2003 and is headquartered in Houston, Texas.

Bio-Path Holdings, Inc. operates as a clinical and preclinical stage oncology focused RNAi nanoparticle drug development company in the United States. The company develops products based on DNAbilize, a drug delivery and antisense technology platform that uses P-ethoxy, which is a deoxyribonucleic acid (DNA) backbone modification intended to protect the DNA from destruction. Its lead drug candidate is prexigebersen, which is in Phase II clinical trials for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome. It is also developing BP1001-A that is in Phase I clinical trial for the treatment of solid tumors; Liposomal Bcl-2 (BP1002), which is in Phase I clinical trial for the treatment of refractory/relapsed lymphoma and chronic lymphocytic leukemia; and Liposomal STAT3 (BP1003) for treating pancreatic cancer, non-small cell lung cancer, and AML. The company was founded in 2007 and is based in Bellaire, Texas.