[Image courtesy of ipopba/Adobe Stock]
The cell and gene therapy sector is poised to deliver a wave of new therapies that could potentially cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023.
While manufacturing and regulatory challenges remain, the cell and gene therapy industry continues to offer significant therapeutic potential and commercial opportunities.
This year, we have beefed up our list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.
In selecting the companies for this list, we weighed public companies’ degree of involvement in cell and gene therapy, including drug developers, equipment vendors and CDMOs specializing in the area.
For private companies, we factored in their funding levels, pipeline and degree of history for the list.
Because the field of cell and gene therapy remains relatively small, this list is a fairly comprehensive overview of companies active in the industry.
| Company | Headquarters | Description | Product examples | Ticker | |
|---|---|---|---|---|---|
| 1 | 4D Molecular Therapeutics | Emeryville, California | 4D Molecular Therapeutics focuses on developing targeted and customizable adeno-associated virus (AAV) vectors for gene therapy. | 4D-125, 4D-110, 4D-310 | Privately-held |
| 2 | Abeona Therapeutics | New York City | Develops gene and cell therapies for rare genetic diseases. | ADVM-022, EB-101, ABO-102 (AAV-SGSH), ABO-101 (AAV-NAGLU) | Nasdaq: ADVM |
| 3 | Abzena | San Diego, California | This CDMO provides integrated services, including cell line development, process and analytical development, and clinical manufacturing. | Develops cell and gene therapies for clients. | Privately-held |
| 4 | Adaptimmune Therapeutics | Abingdon, UK | Pipeline includes SPEAR T-cell therapies for solid tumors. | Afamitresgene autoleucel or “afami-cel”, ADP-A2M4CD8 SPEAR T-cell therapy | Nasdaq: ADAP |
| 5 | Adverum Biotechnologies | Redwood City, California | Developing ADVM-022, a gene therapy for wet AMaryland, a leading cause of blindness in the elderly. | ADVM-022, ADVM-062 | Nasdaq: ADVM |
| 6 | Allogene Therapeutics | South San Francisco, California | Specializes in developing AlloCaliforniaR T-cell therapies for cancer. | ALLO-501 (allogeneic CaliforniaR-T therapy), ALLO-715 (allogeneic CaliforniaR-T therapy) | Nasdaq: ALLO |
| 7 | Alnylam | Cambridge, Massachusetts | Develops RNA interference (RNAi) therapies for rare genetic diseases. | Onpattro (patisiran), Givlaari (givosiran), Oxlumo (lumasiran) | Nasdaq: ALNY |
| 8 | Altor BioScience | Miramar, Florida | Altor has a pipeline of immunotherapies for cancer and infectious diseases. | Develops cell and gene therapies for cancer and inflammatory diseases. | Privately-held |
| 9 | American Gene Technologies | Rockville, Maryland | This gene therapy company specializes in developing treatments for HIV and genetic diseases. | AGT103-T, AGT103-iNKT | Privately-held |
| 10 | Amgen | Thousand Oaks, California | Amgen’s gene therapy and cell therapy products focus on oncology and rare diseases. | Imlygic (talimogene laherparepvec), AMG 714, AMG 592, AMG 420, AMG 701 | Nasdaq: AMGN |
| 11 | Arcellx | Germantown, Maryland | Arcellx specializes in developing ARC T-cell therapies for cancer. | ACLX-001 and ACLX-002 | Nasdaq: ACLX |
| 12 | ArrowHead Pharmaceuticals | Pasadena, California | ArrowHead focuses on developing RNA interference (RNAi) therapies. | ANPDR001, ARO-AAT | Nasdaq: ARWR |
| 13 | Aspen Neuroscience | San Diego, California | Aspen focuses on developing gene therapies for neurodegenerative diseases such as Parkinson’s disease. | AAV-based gene therapies | Privately-held |
| 14 | Astellas Gene Therapies | Boston, Massachusetts | This clinical-stage biotechnology company focuses on developing gene therapies for rare genetic diseases. | Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271 | TYO: 4503 |
| 15 | AstraZeneca/Alexion | Boston, Massachusetts | AstraZeneca focuses on developing gene therapies for rare diseases such as metabolic disorders and lysosomal storage disorders. | Calquence, MEDI3039, MEDI9197, TAVO, LentiGlobin, MEDI9198, Eplontersen | LON: AZN |
| 16 | Atara Biotherapeutics | South San Francisco, California | Focuses on developing T-cell immunotherapies for cancer and autoimmune diseases. | AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06 | Nasdaq: AVRO |
| 17 | Autolus Therapeutics | London | Autolus specializes in developing CaliforniaR-T cell therapies for the treatment of cancer. | Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8 | Nasdaq: AUTL |
| 18 | AVROBIO | Cambridge, Massachusetts | This clinical-stage gene therapy company focused on developing lentiviral-based gene therapies for rare diseases. | EB-101, ABO-102, ABO-50X, AIM Vectors | Nasdaq: ABEO |
| 19 | Axovant Gene Therapies | New York City | Develops gene therapies for neurological and neuromuscular diseases such as Parkinson’s disease and Tay-Sachs disease. | AXO-Lenti-PD, AXO-AAV-GM1, AXO-AAV-GM2, AXO-AAV-OPMaryland | Nasdaq: AXGT |
| 20 | Batavia Biosciences | Leiden, Netherlands | Provides viral vector and mammalian cell line technologies for the development of biopharmaceuticals and vaccines. | Viral Vector and Mammalian Cell Line Technologies | Privately-held |
| 21 | Bayer | Leverkusen, Germany | Has a pipeline of gene therapies for hemophilia and other rare genetic diseases, and has also developed a manufacturing platform for AAV vectors. | Kogenate, Jivi, Hemlibra, Eylea, Kovaltry, Elocta | OTCMKTS: BAYRY |
| 22 | Bean Therapeutics | Boston, Massachusetts | Develops genetic medicines for inherited diseases such as PKU and Friedreich’s ataxia. | Development of Genetic Medicines | Privately-held |
| 23 | BioMarin Pharmaceutical | San Rafael, California | BioMarin develops therapies for rare genetic diseases such as PKU, MPS I, MPS IIIB, Batten disease, and hemophilia A and B. | Brineura, Kuvan, Naglazyme, Palynziq, Vimizim | Nasdaq: BMRN |
| 24 | Bluebird Bio | Cambridge, Massachusetts | Bluebird focuses on developing gene therapies for genetic diseases such as sickle cell anemia, beta-thalassemia and cerebral adrenoleukodystrophy. | Zynteglo, LentiGlobin | Nasdaq: BLUE |
| 25 | BlueRock Therapeutics | Cambridge, Massachusetts | BlueRock specializes in developing induced pluripotent stem cell (iPSC) therapies for cardiovascular, neurology and immunology. | Induced Pluripotent Stem Cell (iPSC) Technology | Privately-held |
| 26 | Bristol-Myers Squibb | New York City | Uses gene editing and cell engineering technologies to develop next-gen immunotherapies for cancer. | Abecma, Onureg, Reblozyl, Zeposia | NYSE: BMY |
| 27 | Cabaletta Bio | Philadelphia | Cabaletta focuses on developing gene therapies for rare autoimmune diseases such as mucosal pemphigus vulgaris and muco-cutaneous pemphigus. | Engineered Autologous T-Cell Therapy | Nasdaq: CaliforniaBA |
| 28 | Capricor Therapeutics | Los Angeles | Capricor develops cell and gene therapies for the treatment of heart disease, including CaliforniaP-1002 and CaliforniaP-2003. | CaliforniaP-1002, CaliforniaP-2003 | Nasdaq: CaliforniaPR |
| 29 | Caribou Biosciences | Berkeley, California | Develops gene editing technologies using CRISPR/Cas9 with a focus on developing therapies for genetic diseases. | CRISPR-Cas9 Gene Editing Technology | Nasdaq: CRBU |
| 30 | Cellares | South San Francisco, California | Focused on automated systems for cell line development and cell line engineering. | Automated systems for cell therapy manufacturing | Privately-held |
| 31 | Cellectis | Paris | Focuses on developing immunotherapies based on gene-edited allogeneic CaliforniaR T-cells. | UCaliforniaRT19, UCaliforniaRT123 | Nasdaq: CLLS |
| 32 | Celularity | Florham Park, New Jersey | Celularity develops allogeneic placental-derived cells for use in cellular therapeutics. | CYNK-001, CYNK-101, COVID-specific immune cells | Nasdaq: CELU |
| 33 | CGT Catapult | London | A not-for-profit organization that helps cell and gene therapy developers scale-up and manufacture their products. | Manufacturing, process development and regulatory support for the cell and gene therapy industry | Non-profit |
| 34 | Charles River Labs | Wilmington, Massachusetts | Supports the development of cell therapies with services including the characterization of cell products, potency assays and biodistribution studies. | Preclinical and clinical research services | NYSE: CRL |
| 35 | Chimeron Bio | Foster City, California | Focuses on developing gene therapies for rare diseases. | CTX001 for blood disorders and sickle cell disease | Privately-held |
| 36 | Coave Therapeutics | San Diego, California | Develops targeted, non-viral gene therapies using its proprietary nanoparticle platform. | Preclinical programs for inherited retinal diseases and cancer immunotherapies | Privately-held |
| 37 | CRISPR Therapeutics | Zug, Switzerland | A gene-editing company specialized in developing transformative gene-based medicines for serious diseases. | CTX001, allogeneic CaliforniaR-T program and therapies for blood stem cell, immuno-oncology and regenerative medicine | Nasdaq: CRSP |
| 38 | Deep Genomics | Toronto, Canada | Uses artificial intelligence to discover and develop genetic therapies. | AI Workbench for RNA biology, and discovery of novel genetic therapies | Privately-held |
| 39 | Dendreon | Seal Beach, California | Develops immune-oncology therapies for the treatment of cancer. | PROVENGE (sipuleucel-T) for prostate cancer | Privately-held |
| 40 | Dyno Therapeutics | Cambridge, Massachusetts | Develops novel gene therapies with its proprietary AI-powered platform. | AAV vectors for gene therapies | Privately-held |
| 41 | Editas Medicine | Cambridge, Massachusetts | The company develops CRISPR-based therapies for genetic diseases. | EDIT-101, EDIT-301, EDIT-201 | Nasdaq: EDIT |
| 42 | eGenesis | Cambridge, Massachusetts | The company’s lead product is a genetically modified pig kidney that is intended for use in humans. | Porcine organs for human transplantation | Privately-held |
| 43 | ElevateBio | Cambridge, Massachusetts | The company’s subsidiaries include AlloVir, a cell therapy company, and HighPassBio, a gene therapy company. | AlloVir, HighPassBio, RegenxBio and others | Privately-held |
| 44 | Eyevensys | Paris, France | Specializes in electro-transfection technology for the sustained intraocular production of therapeutic proteins. | EYS606 for non-infectious uveitis | Privately-held |
| 45 | Fate Therapeutics | San Diego, California | Develops programmed cellular immunotherapies for cancer and immune disorders. | FT516, FT596, FT538 for cancer, FT576 for hematologic malignancies. | Nasdaq: FATE |
| 46 | FinVector | Nottingham, UK | The company’s pipeline includes gene therapies for genetic diseases such as spinal muscular atrophy, and vaccines for diseases such as influenza and COVID-19. | LentiVector platform, including its oncolytic viruses and immune therapies. | Privately-held |
| 47 | Forte Biosciences | South San Francisco, California | The company’s lead product candidate, FB-401, is a topical live biotherapeutic product for the treatment of atopic dermatitis. | FB-401 for atopic dermatitis, FB-201 for acne. | Nasdaq: FBRX |
| 48 | Freeline Therapeutics | Stevenage, UK | Freeline develops gene therapies for inherited diseases using adeno-associated viruses. | FLT180a for Hemophilia B, FLT190 for Fabry disease. | Nasdaq: FRLN |
| 49 | Fresh Tracks Therapeutics | San Francisco, California | The company’s lead product candidate, FT-101, is a gene therapy for phenylketonuria (PKU) that uses the CRISPR/Cas9 gene editing system. | FRTX-02, FRTX-10, FRTX-03 | Nasdaq: FRTX |
| 50 | Gamida Cell | Boston, Massachusetts | The company’s lead product candidate, omidubicel, is for bone marrow transplantation. | Omidubicel | Nasdaq: GMarylandA |
| 51 | GeneQuine Biotherapeutics GmbH | Berlin, Germany | Specializes in developing gene therapies for musculoskeletal disorders. | Gene therapies for osteoarthritis and other musculoskeletal diseases. | Privately-held |
| 52 | Genascence | Palo Alto, California | Aims to treat osteoarthritis with a gene therapy that is injected directly into the affected joint, based on a gene therapy vector carrying a protein, IL-1 receptor antagonist (IL-1Ra) | GNSC-001 | Privately-held |
| 53 | Generation Bio | Cambridge, Massachusetts | The company’s pipeline includes gene therapies for hemophilia A, phenylketonuria and sickle cell disease. | Gene therapies for diseases such as phenylketonuria (PKU), hemophilia A and sickle cell disease. | Nasdaq: GBIO |
| 54 | GenSight Biologics | Paris, France | The company’s pipeline includes gene therapies for Leber hereditary optic neuropathy and retinitis pigmentosa. | LUMEVOQ for Leber Hereditary Optic Neuropathy (LHON) and GS030 for Retinitis Pigmentosa (RP) | EPA: SIGHT |
| 55 | Gilead Sciences | Foster City, California | Gilead subsidiary Kite Pharma focuses on developing CaliforniaR-T cell therapies for cancer. | Yescarta. | Nasdaq: GILD |
| 56 | Graphite Bio | San Francisco, California | The biopharma focuses on developing gene editing therapies to treat genetic diseases. The company’s platform technology allows for the precise editing of genes in vivo. Graphite Bio’s pipeline includes gene therapies for sickle cell disease and other genetic disorders. | Gene therapies for genetic diseases, such as sickle cell disease and genetic blindness. | Nasdaq: GRPH |
| 57 | Homestead BioPharma | Houston | A gene therapy company that focuses on developing therapies for genetic diseases, including cancers. The company’s pipeline includes gene therapies for the treatment of acute myeloid leukemia and other cancers. | Cancer immunotherapies. | Privately-held |
| 58 | Homology Medicines | Bedford, Massachusetts | A gene therapy company that focuses on developing therapies for rare genetic diseases. The company’s pipeline includes gene therapies for phenylketonuria, metachromatic leukodystrophy, and other rare diseases. | Gene therapies for phenylketonuria (PKU) and metachromatic leukodystrophy (MLD). | Nasdaq: FIXX |
| 59 | Intellia Therapeutics | Cambridge, Massachusetts | Specializes in developing gene editing therapies to treat genetic diseases. The company’s pipeline includes gene editing therapies for transthyretin amyloidosis and sickle cell disease, among others. | Pipeline focuses on genetic diseases such as transthyretin amyloidosis (ATTR), hemophilia and sickle cell disease. | Nasdaq: NTLA |
| 60 | Krystal Biotech | Pittsburgh | A gene therapy company that develops and commercializes gene therapies for rare skin diseases. The company’s lead product candidate, KB103, is a gene therapy for dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. | Gene therapies for rare skin diseases, including epidermolysis bullosa (EB). | Nasdaq: KRYS |
| 61 | LogicBio Therapeutics | Cambridge, Massachusetts | Developing a gene editing platform that uses transposons to integrate therapeutic transgenes into the genome without the use of nucleases. The company’s lead programs include treatments for methylmalonic acidemia and phenylketonuria. | LB-001 for methylmalonic acidemia and LB-10 for phenylketonuria | Nasdaq: LOGC |
| 62 | Lonza | Basel, Switzerland | Lonza provides a wide range of contract manufacturing and development services to the pharmaceutical and biotech industries. The company specializes in the production of biologics and cell therapies. | Contract manufacturing and development services for biologics and cell therapies | SWX: LONN |
| 63 | Lysogene | Paris, France | Lysogene is focused on developing gene therapies for rare central nervous system (CNS) diseases. The company’s lead program is a gene therapy for the treatment of Sanfilippo syndrome type A. | LYS-SAF302 for Sanfilippo syndrome type A | LON: 0RNL |
| 64 | MaxCyte | Gaithersburg, Maryland | MaxCyte provides a platform for high-performance cell engineering. The company’s Flow Electroporation technology enables the rapid and efficient modification of cells for use in a variety of applications, including cell therapy, gene editing, and biomanufacturing. | Flow Electroporation technology for high-performance cell engineering | LON: MXCT |
| 65 | MeiraGTx | London | MeiraGTx is developing gene therapies for rare genetic diseases, with a focus on diseases of the eye and salivary gland. The company’s lead program is a gene therapy for the treatment of achromatopsia. | AAV-CNGB3 for achromatopsia | Nasdaq: MGTX |
| 66 | Mesoblast Limited | Melbourne, Australia | Mesoblast is developing allogeneic cell therapies for the treatment of inflammatory and immune-mediated diseases. The company’s lead product is a mesenchymal stem cell therapy for the treatment of graft versus host disease (GvHD). | Remestemcel-L for GvHD | ASX: MSB |
| 67 | Metagenomi | Emeryville, California | Metagenomi is developing a platform for the discovery and development of novel CRISPR systems. The company’s lead program is a novel CRISPR system for the treatment of genetic diseases. | Novel CRISPR system for the treatment of genetic diseases | privately held |
| 68 | Mission Bio | South San Francisco, California | Mission Bio is developing a platform for single-cell genomics. The company’s Tapestri platform enables the analysis of individual cells in complex biological samples, such as tumors. | Tapestri platform for single-cell genomics | privately held |
| 69 | NexImmune | Gaithersburg, Maryland | NexImmune is developing cell therapies for the treatment of cancer and autoimmune diseases. The company’s lead product is a T cell therapy for the treatment of acute myeloid leukemia (AML). | NEXI-001 for AML | Nasdaq: NEXI |
| 70 | NextCure | Beltsville, Maryland | NextCure is focused on discovering and developing next-generation immuno-oncology products. | NC318, NC410 | Nasdaq: NXTC |
| 71 | Nightstar Therapeutics | London | Nightstar Therapeutics is developing gene therapies for inherited retinal diseases. | NSR-REP1, NSR-RPGR | Privately-held |
| 72 | Novartis AG (Was AveXis) | Basel, Switzerland | Novartis AG (formerly AveXis) is focused on developing gene therapies for rare genetic diseases. | Zolgensma | NYSE: NVS |
| 73 | Orchard Therapeutics | London | Specializes in gene therapies for rare diseases, with a focus on primary immune deficiencies, metabolic disorders, and inherited blood diseases. | Strimvelis, OTL-101 | Nasdaq: ORTX |
| 74 | Oxford Biomedica | Oxford, UK | Developeslentiviral vector-based gene therapies for rare and life-threatening diseases, including cancer and Parkinson’s disease. | LentiVector platform | LON: OXB |
| 75 | Passage Bio | Philadelphia | The company’s technology platform enables the precise editing of genes in vivo. | PBGM01, PBFT02 | Nasdaq: PASG |
| 76 | Pluristem Therapeutics | Haifa, Israel | Pluristem Therapeutics is focused on improving the health and well-being of patients by developing innovative cell therapies that can transform the treatment of serious diseases. | PLX-R18, PLX-PAD | Nasdaq: PSTI |
| 77 | Poseida Therapeutics | San Diego, California | The company’s technology platform allows for the precise editing of genes in vivo, enabling the development of gene therapies that can address the root cause of genetic diseases. | P-BCMassachusetts-ALLO1, P-PSMassachusetts-101 | Nasdaq: PSTX |
| 78 | Precision BioSciences | Durham, North Carolina | The company’s pipeline includes gene editing therapies for genetic diseases such as sickle cell disease and cancer immunotherapies. | ARCUS genome editing platform | Nasdaq: DTIL |
| 79 | Regenxbio Inc. | Rockville, Maryland | Regenxbio specializes in the development of AAV gene therapies for a range of diseases. | RGX-121, RGX-314 | Nasdaq: RGNX |
| 80 | Roche Holding AG | Basel, Switzerland | Roche has a significant presence in the field of cell and gene therapy. It has a number of partnerships and collaborations with companies working in the space. | Roche has several ongoing programs in the field of cell and gene therapy, including for hemophilia A, Huntington’s disease, spinal muscle atrophy, CaliforniaR-T cell therapy and ophthalmology. | SWX: ROG |
| 81 | Rocket Pharmaceuticals | New York City | This clinical-stage company is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. | RP-A501, RP-L102, RP-A201, RP-A301 | Nasdaq: RCKT |
| 82 | Sangamo Therapeutics | Brisbane, California | The company develops genomic therapies for cancer, monogenic diseases, and infectious diseases. | SB-525, ST-400, SB-318, ST-920, TX200, BIVV003, ST-101, KITE-037 | Nasdaq: SGMO |
| 83 | Sarepta Therapeutics Inc. | Cambridge, Massachusetts | The company develops treatments for rare neuromuscular diseases, including Duchenne muscular dystrophy (DMaryland), Limb-girdle muscular dystrophy (LGMaryland), and Charcot-Marie-Tooth (CMT). | Exondys 51, Vyondys 53, golodirsen, casimersen, SRP-9001, SRP-9003, SRP-5051, SRP-707, SRP-9004 | Nasdaq: SRPT |
| 84 | Solid Biosciences | Cambridge, Massachusetts | The company’s lead product candidate is a microdystrophin gene therapy that aims to restore functional dystrophin protein expression in patients with DMaryland. | SGT-001 | Nasdaq: SLDB |
| 85 | SQZ Biotechnologies | Watertown, Massachusetts | Develops cellular therapies for cancer, infectious diseases, and other serious conditions. The company’s platform technology allows for the rapid engineering of cells for use in cell therapy. | SQZ-PBMC-HPV for the treatment of human papillomavirus-associated tumors and SQZ-AAC for the treatment of solid tumors | NYSE: SQZ |
| 86 | StrideBio | Durham, NC | Develops viral vector technologies for gene therapy. | STR101, STR102, STR103, | Privately-held |
| 87 | Suzhou Ribo Life Science | Suzhou, China | The company’s pipeline includes RNA therapies for cancer and infectious diseases. | The company has eight products in development, including assets for hepatitis B and non-arteritic anterior ischemic optic neuropathy (NAION). | Privately-held |
| 88 | Takeda Pharmaceutical | Tokyo, Japan | Focuses on developing cell and gene therapies addressing significant unmet medical needs in oncology, rare genetic diseases and neurological disorders. | Adynovate, Ninlaro, Alunbrig, Gattex/Revestive, Takhzyro, Natpara, Firazyr, Iclusig, Velcade | NYSE: TAK |
| 89 | Thermo Fisher Scientific | Waltham, Massachusetts | Thermo Fisher offers tools for gene editing, including CRISPR/Cas9 and TALEN technologies, as well as a range of gene synthesis and molecular biology tools. | Ion Torrent, Applied Biosystems, Invitrogen, Gibco, Molecular Probes, TaqMan | NYSE: TMO |
| 90 | Tmunity Therapeutics | Philadelphia | Develops T cell therapies for the treatment of cancer and other diseases. | Tmunity CD19, Tmunity NY-ESO, Tmunity WT1, Tmunity EGFRvIII | Privately-held |
| 91 | TriLink BioTechnologies (Maravai LifeSciences) | San Diego, California | TriLink BioTechnologies provides custom oligonucleotides, mRNA and gene editing enzymes to the life science research, diagnostic, and therapeutic markets. | mRNA vaccines, mRNA therapeutics, CRISPR/Cas9 gene editing and long non-coding RNA | Nasdaq: MRVI |
| 92 | Ultragenyx | Novato, California | The company’s pipeline includes therapies for genetic diseases, metabolic diseases and rare cancers. | Dojolvi, Crysvita, Mepsevii | Nasdaq: RARE |
| 93 | UniQure | Lexington, Massachusetts | UniQure’s technology platform allows for the targeted delivery of therapeutic genes to specific cells. | AMT-130, AMT-150, AMT-190 | Nasdaq: QURE |
| 94 | Veneti Inc. | South San Francisco, California | The company’s pipeline includes cell therapies for hematologic malignancies and solid tumors. | VET-001 (acute myeloid leukemia), VET-002 (solid tumors), VET-003 (solid tumors) | Privately-held |
| 95 | Vericel | Cambridge, Massachusetts | The company’s product portfolio includes cell therapies for cartilage repair and burns. | MassachusettsCI (cartilage regeneration), Epicel (skin regeneration) | Nasdaq: VCEL |
| 96 | Vertex Pharmaceuticals | Boston, Massachusetts | Specializes in the development of small molecule drugs for the treatment of serious diseases, including cystic fibrosis, sickle cell disease, and beta thalassemia. | Trikafta (cystic fibrosis), Kaftrio (cystic fibrosis), Symdeko (cystic fibrosis) | Nasdaq: VRTX |
| 97 | Verve Therapeutics | Cambridge, Massachusetts | The company’s technology platform allows for the precise editing of genes in vivo. | Verve’s lead programs (targets undisclosed) | Nasdaq: VERV |
| 98 | Vivet Therapeutics | Paris, France | The company’s lead program is a gene therapy for Wilson disease, a rare genetic disorder that causes the accumulation of copper in the liver and other organs. | VTX-801, VTX-803 | Privately-held |
| 99 | Vizgen | Woburn, Massachusetts | The company’s technology platform allows for the simultaneous detection of multiple transcripts within single cells. | MERFISH (Multiplexed Error-Robust Fluorescence In Situ Hybridization) | Privately-held |
| 100 | Voyager Therapeutics | Cambridge, Massachusetts | Focused on developing gene therapies for neurological diseases, including Parkinson’s disease, Huntington’s disease and ALS. | VY-HTT01, VY-SOD102, VY-AADC01 | Nasdaq: VYGR |