The cell and gene therapy sector is poised to deliver a wave of new therapies with the potential to 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.
We have beefed up our 2022 list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.
In selecting the companies for the latest list, we weighed public companies’ degree of involvement in cell and gene therapy, focusing primarily on including drug developers 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.
We have a separate list of cell and gene therapy vendors and suppliers here.
| Company | Headquarters | Description | Product/pipeline examples | |
|---|---|---|---|---|
| 1 | 4D Molecular Therapeutics | Emeryville, California | Focuses on developing targeted and customizable adeno-associated virus (AAV) vectors for gene therapy. | 4D-125, 4D-110, 4D-310 |
| 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) |
| 3 | Adaptimmune Therapeutics | Abingdon, UK | Pipeline includes SPEAR T-cell therapies for solid tumors. | Afamitresgene autoleucel or “afami-cel”, ADP-A2M4CD8 SPEAR T-cell therapy |
| 4 | Adverum Biotechnologies | Redwood City, California | Developing ADVM-022, a gene therapy for wet AMD, a leading cause of blindness in the elderly. | ADVM-022, ADVM-062 |
| 5 | Allogene Therapeutics | South San Francisco, California | Specializes in developing allogeneic CAR-T products for cancer | ALLO-501, ALLO-715 |
| 6 | Alnylam | Cambridge, Massachusetts | Develops RNA interference (RNAi) therapies for rare genetic diseases. | Onpattro (patisiran), Givlaari (givosiran), Oxlumo (lumasiran) |
| 7 | 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 |
| 8 | American Gene Technologies | Rockville, Maryland | This gene therapy company specializes in developing treatments for HIV and genetic diseases. | AGT103-T, AGT103-iNKT |
| 9 | Amgen | Thousand Oaks, California | Amgen’s gene therapy and cell therapy products focus on oncology and rare diseases. | AMG 510, AMG 330, AMG 596, AMG 757, AMG 673, AMG 119, AMG 592 |
| 10 | Arcellx | Germantown, Maryland | Arcellx specializes in developing ARC T-cell therapies for cancer. | ACLX-001 and ACLX-002 |
| 11 | ArrowHead Pharmaceuticals | Pasadena, California | ArrowHead focuses on developing RNA interference (RNAi) therapies. | ANPDR001, ARO-AAT |
| 12 | Aspen Neuroscience | San Diego, California | Aspen focuses on developing gene therapies for neurodegenerative diseases such as Parkinson’s disease. | AAV-based gene therapies |
| 13 | 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 |
| 14 | AstraZeneca/Alexion | Boston, Massachusetts | Develops gene therapies for rare diseases such as metabolic disorders and lysosomal storage disorders. | LentiGlobin, MEDI9197, MEDI9198 |
| 15 | Atara Biotherapeutics | South San Francisco, California | Focuses on developing T-cell immunotherapies for cancer and autoimmune diseases. | ATA188, ATA2271, ATA3271, ATA3219 |
| 16 | Autolus Therapeutics | London | Autolus specializes in developing CAR-T cell therapies for the treatment of cancer. | Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8 |
| 17 | AVROBIO | Cambridge, Massachusetts | Focuses on developing lentiviral-based gene therapies for rare diseases. | AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05 |
| 18 | Axovant Gene Therapies | New York City | Develops gene therapies for neurological and neuromuscular diseases such as Parkinson’s and Tay-Sachs disease. | AXO-Lenti-PD, AXO-AAV-GM1, AXO-AAV-GM2, AXO-AAV-OP |
| 19 | 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 |
| 20 | Bayer | Leverkusen, Germany | Bayer’s gene therapy efforts focus on hemophilia and other rare genetic diseases. It has also developed a manufacturing platform for AAV vectors. | BAY 2599023, BAY 2599023 + emicizumab, BAY 2599023 + BMN 307, BAY 2599023 + etranacogene dezaparvovec |
| 21 | Bean Therapeutics | Boston, Massachusetts | Develops genetic medicines for inherited diseases such as PKU and Friedreich’s ataxia. | Development of genetic medicines |
| 22 | BioMarin Pharmaceutical | San Rafael, California | BioMarin develops therapies for rare genetic diseases such as PKU, MPS I, MPS IIIB, Batten disease and hemophilia. | Brineura, Kuvan, Naglazyme, Palynziq, Vimizim |
| 23 | Bluebird Bio | Cambridge, Massachusetts | Develops gene therapies for genetic diseases such as sickle cell anemia, beta-thalassemia and cerebral adrenoleukodystrophy. | Zynteglo, LentiGlobin |
| 24 | 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 |
| 25 | Bristol-Myers Squibb | New York City | Uses gene editing and cell engineering technologies to develop next-gen immunotherapies for cancer. | BMS-986249, BMS-986253, BMS-986258, Liso-cel (JCAR017), bb21217, bb21222 |
| 26 | Cabaletta Bio | Philadelphia | Develops gene therapies for rare autoimmune diseases such as mucosal pemphigus vulgaris and muco-cutaneous pemphigus. | Engineered Autologous T-cell therapy |
| 27 | Capricor Therapeutics | Los Angeles | Capricor develops cell and gene therapies for heart disease. | CAP-1002, CAP-1001, CAP-2003, CAP-3001 |
| 28 | Caribou Biosciences | Berkeley, California | Develops gene editing technologies using CRISPR/Cas9 with a focus on developing therapies for genetic diseases. | Pipeline of off-the-shelf genome-edited CAR-T and CAR-NK cell therapies |
| 29 | Celgene (now Bristol-Myers Squibb) | Summit, New Jersey | This Bristol Myers Squibb company has developed a CAR-T cell therapy that recognizes the B-cell maturation antigen (BCMA) and kills BCMA-expressing tumor cells. | Abecma (idecabtagene vicleucel) |
| 30 | Cellectis | Paris | Focuses on developing immunotherapies based on gene-edited allogeneic CAR T-cells. | UCART19, UCART22, UCART123, ALLO-501, ALLO-715 |
| 31 | Celularity | Florham Park, New Jersey | Celularity develops allogeneic placental-derived cells for use in cellular therapeutics. | CYNK-001, CYNK-101, COVID-specific immune cells |
| 32 | Celyad Oncology | Mont-Saint-Guibert, Belgium | Develops chimeric antigen receptor T cell (CAR T) therapies for cancer. | CYAD-101, CYAD-211, CYAD-02 |
| 33 | Chimeron Bio | Foster City, California | Focuses on developing gene therapies for rare diseases. | CB-101, ChaESAR RNA therapy |
| 34 | 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 |
| 35 | CRISPR Therapeutics | Zug, Switzerland | A gene-editing company specialized in developing transformative gene-based medicines for serious diseases. | CTX001 and therapies for blood stem cell, immuno-oncology and regenerative medicine |
| 36 | CSL Behring | King of Prussia, Pennsylvania | Focuses on adeno-associated virus (AAV)-based gene therapy and stem-cell gene therapies for primary immunodeficiency diseases. | Hemgenix (etranacogene dezaparvovec) |
| 37 | Deep Genomics | Toronto, Canada | AI drug development company’s focus includes the discovery and development of genetic therapies. | AI Workbench for RNA biology and discovery of novel genetic therapies |
| 38 | Dendreon | Seal Beach, California | Develops immune-oncology therapies for the treatment of cancer. | PROVENGE (sipuleucel-T) for prostate cancer |
| 39 | Dyno Therapeutics | Cambridge, Massachusetts | Develops novel gene therapies with its proprietary AI-powered platform. | AAV vectors for gene therapies |
| 40 | Editas Medicine | Cambridge, Massachusetts | The company develops CRISPR-based therapies for genetic diseases. | EDIT-101, EDIT-301, EDIT-201 |
| 41 | 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 |
| 42 | ElevateBio | Cambridge, Massachusetts | The firm’s subsidiaries include AlloVir, a cell therapy company and HighPassBio, a gene therapy company. | AlloVir, HighPassBio, RegenxBio and others |
| 43 | Enzyvant | Durham, North Carolina | Company scored FDA approval for Rethymic, is a regenerative tissue therapy for the ultra-rare condition of congenital athymia. | Rethymic (allogeneic processed thymus tissue-agdc) |
| 44 | Eyevensys | Paris, France | Specializes in electro-transfection technology for the sustained intraocular production of therapeutic proteins. | EYS606 for non-infectious uveitis |
| 45 | Fate Therapeutics | San Diego, California | Develops programmed cellular immunotherapies for cancer and immune disorders. | FT516, FT596, FT538 for cancer, FT576 |
| 46 | Ferring Pharmaceuticals | Saint-Prex, Switzerland | The company has won FDA approval for Adstiladrin, is a novel adenovirus vector-based gene therapy, for adults with high-risk, Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer NMIBC) with carcinoma in situ. | Adstiladrin (nadofaragene firadenovec-vncg) |
| 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 |
| 48 | Freeline Therapeutics | Stevenage, UK | Freeline develops gene therapies for inherited diseases using adeno-associated viruses. | FLT180a for Hemophilia B, FLT190 for Fabry disease |
| 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 |
| 50 | Gamida Cell | Boston, Massachusetts | The company’s lead product candidate, omidubicel, is for bone marrow transplantation. | Omidubicel |
| 51 | GeneQuine Biotherapeutics GmbH | Berlin, Germany | Specializes in developing gene therapies for musculoskeletal disorders. | Gene therapies for osteoarthritis and other musculoskeletal diseases |
| 52 | Genascence | Palo Alto, California | Aims to treat osteoarthritis with a gene therapy that is injected directly into the affected joint. | GNSC-001 |
| 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 |
| 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) |
| 55 | Gilead Sciences | Foster City, California | Gilead subsidiary Kite Pharma focuses on developing CaliforniaR-T cell therapies for cancer. | Yescarta |
| 56 | Graphite Bio | San Francisco, California | 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 |
| 57 | Homestead BioPharma | Houston | The company’s pipeline includes gene therapies for the treatment of acute myeloid leukemia and other cancers. | Cancer immunotherapies. |
| 58 | Homology Medicines | Bedford, Massachusetts | A gene therapy company that focuses on developing therapies for rare genetic diseases. | Gene therapies for phenylketonuria (PKU) and metachromatic leukodystrophy (MLD). |
| 59 | Intellia Therapeutics | Cambridge, Massachusetts | The company’s pipeline includes gene editing therapies for transthyretin amyloidosis and sickle cell disease. | Pipeline focuses on genetic diseases such as transthyretin amyloidosis (ATTR), hemophilia and sickle cell disease |
| 60 | Juno Therapeutics | Seattle | Develops cell-based immunotherapies based on its chimeric antigen receptor and T cell receptor technologies to genetically engineer T cells for oncology indications. | Lisocabtagene maraleucel (Breyanzi) |
| 61 | Krystal Biotech | Pittsburgh | 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) |
| 62 | LogicBio Therapeutics | Cambridge, Massachusetts | Developing a gene editing platform that uses transposons to integrate therapeutic transgenes into the genome without the use of nucleases. | LB-001 for methylmalonic acidemia and LB-10 for phenylketonuria |
| 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 |
| 64 | MeiraGTx | London | MeiraGTx is developing gene therapies for rare genetic diseases, with a focus on diseases of the eye and salivary gland. | AAV-CNGB3 for achromatopsia |
| 65 | Mesoblast | Melbourne, Australia | The company’s lead product is a mesenchymal stem cell therapy for the treatment of graft versus host disease (GvHD). | Remestemcel-L for GvHD |
| 66 | 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. The company has attracted $275M in Series B financing to progress their lead therapeutic programs into the clinic. | The company has an internal and partnered pipeline of in vivo genetic medicines. Scant information is available about them, however. |
| 67 | 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 |
| 68 | NextCure | Beltsville, Maryland | NextCure is focused on discovering and developing next-generation immuno-oncology products. | NC318, NC410 |
| 69 | Nightstar Therapeutics | London | Nightstar Therapeutics is developing gene therapies for inherited retinal diseases. | NSR-REP1, NSR-RPGR |
| 70 | Novartis | Basel, Switzerland | Novartis AG (formerly AveXis) is focused on developing gene therapies for rare genetic diseases. | Zolgensma |
| 71 | 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 |
| 72 | Oxford Biomedica | Oxford, UK | Develops lentiviral vector-based gene therapies for rare and life-threatening diseases, including cancer and Parkinson’s disease. | LentiGlobin, OXB-102, OXB-202, AXO-Lenti-PD, OXB-301 |
| 73 | Passage Bio | Philadelphia | The company’s technology platform enables the precise editing of genes in vivo. | PBGM01, PBFT02 |
| 74 | Pfizer | New York City | Focused on diseases with single-gene defects, including certain neuromuscular and hematologic diseases. | VTX-801 |
| 75 | 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 |
| 76 | 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-BCMA-ALLO1, P-PSMA-101 |
| 77 | Regeneron | Tarrytown, New York | The company’s focus includes developing therapies for genetic diseases caused by a single gene mutation. | RGN-352, RGX-121, REGN1979, REGN5458, REGN5459, REGN5678 |
| 78 | Regenxbio | Rockville, Maryland | Regenxbio specializes in the development of AAV gene therapies for a range of diseases. | RGX-121, RGX-314 |
| 79 | ReNeuron | Bridgend, UK | ReNeuron’s core focus is on its CustomEx exosome technology platform. | ReN0001, ReN0000 |
| 80 | Roche | 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. | Cell and gene therapy programs for hemophilia A, Huntington’s disease, spinal muscle atrophy and ophthalmology |
| 81 | Rocket Pharmaceuticals | New York City | Advancing an integrated pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. | RP-A501, RP-L102, RP-A201, RP-A301 |
| 82 | Rubius Therapeutics | Cambridge, Massachusetts | Company is genetically engineering red blood cells to develop a new class of cellular medicines called Red Cell Therapeutics. | RTX-240, RTX-224, RTX-T1D |
| 83 | Sana Biotechnology | Seattle | Working to repair and control genes in cells or replace missing or damaged cells to address the underlying cause of diseases. Sana is building differentiated capabilities across the spectrum of cell and gene therapy | SG295, SG242, SG221, SG239, SG328 |
| 84 | 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 |
| 85 | Sarepta Therapeutics | Cambridge, Massachusetts | Develops treatments for rare neuromuscular diseases, including Duchenne muscular dystrophy, Limb-girdle muscular dystrophy and Charcot-Marie-Tooth. | Exondys 51, Vyondys 53, golodirsen, casimersen, SRP-9001, SRP-9003, SRP-5051, SRP-707, SRP-9004 |
| 86 | Solid Biosciences | Cambridge, Massachusetts | The company’s lead product candidate is a microdystrophin gene therapy that aims to restore functional dystrophin protein expression in Duchenne muscular dystrophy. | SGT-001 |
| 87 | Spark Therapeutics | Philadelphia | Specializes in adeno-associated viral (AAV) vectors for debilitating genetic diseases. | Luxturna (voretigene neparvovec-rzyl) |
| 88 | SQZ Biotechnologies | Watertown, Massachusetts | Develops cellular therapies for cancer, infectious diseases and other serious conditions. | SQZ-PBMC-HPV for human papillomavirus-associated tumors and SQZ-AAC for solid tumors |
| 89 | StrideBio | Durham, NC | Develops viral vector technologies for gene therapy. | STR101, STR102, STR103 |
| 90 | Suzhou Ribo Life Science | Suzhou, China | The company’s pipeline includes RNA therapies for cancer and infectious diseases. | Eight products in development include assets for hepatitis B and non-arteritic anterior ischemic optic neuropathy |
| 91 | 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 |
| 92 | Tmunity Therapeutics | Philadelphia | Develops T cell therapies for the treatment of cancer and other diseases. | Tmunity CD19, Tmunity NY-ESO, Tmunity WT1, Tmunity EGFRvIII |
| 93 | Ultragenyx | Novato, California | The company’s pipeline includes therapies for genetic diseases, metabolic diseases and rare cancers. | Dojolvi, Crysvita, Mepsevii |
| 94 | UniQure | Lexington, Massachusetts | UniQure’s technology platform allows for the targeted delivery of therapeutic genes to specific cells. | AMT-130, AMT-150, AMT-190 |
| 95 | Veneti | 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) |
| 96 | Vericel | Cambridge, Massachusetts | The company’s product portfolio includes cell therapies for cartilage repair and burns. | MACI (cartilage regeneration), Epicel (skin regeneration) |
| 97 | 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) |
| 98 | Verve Therapeutics | Cambridge, Massachusetts | The company’s technology platform allows for the precise editing of genes in vivo. | VERVE-101, VERVE-201 |
| 99 | 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 |
| 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 |
Filed Under: Cell & gene therapy, Pharma 50, Special Feature
