Pharma50: 50 Leading Cell and gene therapy companies
Cell and gene therapies promise to enable broad changes in the healthcare system over the next decade, prompting a growing number of cell and gene therapy companies to join the space.
Consider that a cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients. Cell and gene therapy companies also aim to cure common forms of blindness and restore sight in patients with inherited retinal diseases. In addition, RNA has potential in treating brain cancer, as Mass General Brigham observes in its list of one dozen potentially disruptive cell and gene therapy innovations.
The field continues to multiply in size. According to the market research firm Emergent Research, international cell and gene therapy companies could generate $6.6 billion in revenue by 2027, with a projected CAGR of 19.8% from 2020 to 2027.
[Check out our updated list featuring 100 cell and gene therapy companies.]
Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies.
| Top cell and gene therapy companies | Company description | Product/product candidate examples | Ticker | |
| 1 | Adaptimmune Therapeutics | Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. | Afamitresgene autoleucel or “afami-cel” (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy | Nasdaq: ADAP |
| 2 | AVROBIO | AvroBio focuses on lyosomal disorders. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. | AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06 | Nasdaq: AVRO |
| 3 | Abeona Therapeutics | Abeona aims to develop therapies for a variety of diseases, including Recessive Dystrophic Epidermolysis Bullosa and Sanfilippo Syndrome Type A. | EB-101, ABO-102, ABO-50X, AIM Vectors | Nasdaq: ABEO |
| 4 | Adverum Biotechnologies | Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. | ADVM-022 | Nasdaq: ADVM |
| 5 | Alnylam | Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. The company has scored approvals for several RNAi therapeutics. | ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), AMVUTTRA (vutrisiran) | Nasdaq: ALNY |
| 6 | ArrowHead Pharmaceuticals | Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. | ARO-AAT, ARO-APOC3, ARO-ANG3, ARO-HSD, ARO-C3 | Nasdaq: ARWR |
| 7 | Aspen Neuroscience | The company is using a stem cell biology and genomics platform to develop a novel autologous induced pluripotent stem cell (iPSC)-derived neuron replacement therapy for Parkinson’s Disease. | ANPD001, ANPD002 | privately held |
| 8 | Astellas Gene Therapies | In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. | AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. | TYO: 4503 |
| 9 | Atara Biotherapeutics | Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. | Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271 | Nasdaq: ATRA |
| 10 | Autolus Therapeutics | Autolus specializes in developing CAR-T cell therapies. The company was spun-out from University College London in 2014. The company’s lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. | Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8 | Nasdaq: AUTL |
| 11 | Bayer | Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. That year, Bayer also acquired BlueRock Therapeutics. In late 2020, the company completed the acquisition of Asklepios Biopharmaceutical to bolster the unit. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. | Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinson’s disease gene therapy, Parkinson’s disease cell therapy | OTCMKTS: BAYRY |
| 12 | Bluebird Bio | The biotech specializes in creating gene therapies for severe genetic disorders and cancer. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. | eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel | Nasdaq: BLUE |
| 13 | Bristol-Myers Squibb | The company has two approved chimeric antigen receptor (CAR T) cell therapies in hematologic malignancies that use two distinct targets targeting separate blood cancers. | Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel) | NYSE: BMY |
| 14 | Cabaletta Bio | Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. | DSG3-CAART | Nasdaq: CABA |
| 15 | Caribou Biosciences | The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. In September 2021, the company announced positive CRISPR hybrid RNA-DNA data. | CB-010, CB-011, CB-012, CB-020 | Nasdaq: CRBU |
| 16 | Capricor Therapeutics | The biotech is developing novel cell and exosome-based therapeutics. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. | CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes | Nasdaq: CAPR |
| 17 | Cellares | The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its Cell Shuttle, which it dubs a “factory in a box.” | Cell Shuttle manufacturing platform | Privately held |
| 18 | Cellectis | Cellectis has more than two decades of experience in gene editing. The company’s core focus areas include immuno-oncology and plant sciences. | UCART123, UCART22, UCARTCS1, UCART19, ALLO-501, ALLO-715 | Nasdaq: CLLS |
| 19 | Cellular Biomedicine Group | The company is developing novel cell therapies for oncology and degenerative diseases. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. | anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T | Cellular Biomedicine Group |
| 20 | Celularity | Focuses on allogeneic placental-derived cells. The company has multiple cell therapy candidates in pre-clinical and clinical development. | CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002 | Nasdaq: CELU |
| 21 | CRISPR Therapeutics | Specializing in CRISPR/Cas9 technology, CRISPR Therapeutics is initially targeting the blood diseases β-thalassemia and sickle cell disease. The company’s allogeneic CAR-T program targets B-cell malignancies. It is also developing therapies for blood stem cell, immuno-oncology and regenerative medicine. | exa-cel, CTX110, CTX112, CTX130, CTX131, anti-CD83 autologous CAR-T, VCTX210, VCTX211, VCTX212, CTX310 | Nasdaq: CRSP |
| 22 | Deep Genomics | The company specializes in the use of AI to build novel genetic therapies. The company boasts that its AI Workbench can help manage the complexity in RNA biology while identifying novel targets. | The company’s AI workbench is finding use for CNS and metabolic disease. The company also has a collaboration with BioMarin. | privately held |
| 23 | Editas Medicine | The company is working with the Cas9 and Cas12a CRISPR nucleases. The two nucleases give it access to a variety of genetic mutations and develop targeted and durable gene edited medicines. | EDIT-101, EDIT-103, EDIT-102, EDIT-202, EDIT-301 | Nasdaq: EDIT |
| 24 | eGenesis | The biotech has developed a multiplex gene editing and genome engineering platform for applications in solid organ and therapeutic cell transplantation. | HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion | privately held |
| 25 | Freeline Therapeutics | eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. | FLT180a, FLT190, FLT201 | Nasdaq: FRLN |
| 26 | Gamida Cell | The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. It has a diverse approach to cellular therapy using nicotinamide (NAM) to expand multiple cell types. | omidubicel, GDA-20, GDA-301, GDA-401, GDA-501, GDA-601 | Nasdaq: GMDA |
| 27 | Generation Bio | Generation Bio has developed a platform with a ceDNA construct, ctLNP delivery system and scalable rapid enzymatic manufacturing process. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. | ceDNA, ctLNP | Nasdaq: GBIO |
| 28 | Graphite Bio | The gene editing company focuses on diseases for patients with serious diseases. Graphite Bio is building on CRISPR technology and working with the cell’s natural DNA repair processes to rewrite genes. | GPH101, GPH102 | Nasdaq: GRPH |
| 29 | Intellia Therapeutics | The company is developing CRISPR/Cas9 genome editing technology. It has a pipeline of in vivo and ex vivo therapies. | NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001 | Nasdaq: NTLA |
| 30 | Krystal Biotech | Krystal Biotech specializes in redosable gene therapy. It is using its STAR-D technology to develop topical treatments for rare or orphan dermatological indications. | B-VEC, KB105, KB104, KB407, KB408, KB301 | Nasdaq: KRYS |
| 31 | MaxCyte | Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. The platform supports the engineering of almost all cell types, including human primary cells and with any molecule. | The company sells a variety of instruments and consumables. | LON: MXCT |
| 32 | MeiraGTx | Focuses on clinical-stage gene therapy. It has six programs in clinical development. Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinson’s disease. | AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006 | Nasdaq: MGTX |
| 33 | Mesoblast Limited | The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. | The company’s late-stage clinical pipeline is targeting acute graft versus host disease, inflammatory bowel disease, acute respiratory distress syndrome, chronic low back pain and chronic heart failure reduced ejection fraction. | ASX: MSB |
| 34 | Metagenomi | The company is pursuing various applications for metabolic, genetic diseases and oncology with its gene editing technologies. | The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. | privately held |
| 35 | Mission Bio | The company’s Tapestri platform can simultaneously provide genotype and phenotype data from the same cell across thousands of single cells. | The company’s multi-omics approach supports the development of cell and gene therapies. | privately held |
| 36 | NexImmune | The company’s NEXI-001 and NEXI-002 programs are in Phase 1/2 clinical studies for relapsed acute myeloid leukemia after allogeneic stem cell transplantation and multiple myeloma refractory to >3 prior lines of therapy, respectively. | NEXI-001, NEXI-002 | Nasdaq: NEXI |
| 37 | NextCure | The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. | NC318, NC410, NC762, NC525 | Nasdaq: NXTC |
| 38 | Novartis AG (Was AveXis) | Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. In 2020, the company renamed the previously acquired AveXis to Novartis Gene Therapies. | Zolgensma (onasemnogene abeparvovec) | NYSE: NVS |
| 39 | Passage Bio | Focusing on developing therapeutics for disorders of the central nervous system. The company’s pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. | PBGM01, PBKR03, PBML04, PBFT02 | Nasdaq: PASG |
| 40 | Pluristem Therapeutics | The clinical-stage regenerative medicine company specializes in using placental cells and proprietary, 3-D technology platform to develop cell therapies for inflammation, muscle injuries, hematological disorders and radiation exposure. | PLX-PAD, PLX-R18 | Nasdaq: PSTI |
| 41 | Regenxbio Inc. | The company’s gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. It also selectively licenses its NAV vectors to other biotechnology companies. | RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381 | Nasdaq: RGNX |
| 42 | Roche Holding AG | Roche’s cell and gene therapy efforts gained steam in 2021 when the company announced a partnership with Shape Therapeutics related to the development of potential treatments for Alzheimer’s, Parkinson’s and rare diseases. The collaboration could be worth more than $3 billion. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. | Luxturna (voretigene neparvovec-rzyl) | SWX: ROG |
| 43 | Rocket Pharmaceuticals | Focuses on developing novel gene therapies for rare inherited genetic diseases. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. Rocket Pharmaceuticals is aiming for its first regulatory filing in H1 of 2023 for its LVV gene therapy RP-201 for Leukocyte Adhesion Deficiency-I (LAD-I) a rare, autosomal recessive pediatric disease where, without a successful bone marrow transplant. | AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301 | Nasdaq: RCKT |
| 44 | Sangamo Therapeutics | The company has a variety of gene and cell therapy programs in the clinic and preclinical programs in genome engineering and off-the-shelf cell therapy. | Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136 | Nasdaq: SGMO |
| 45 | Sarepta Therapeutics Inc. | The biotech aims to engineer precision genetic medicine for rare diseases. | Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004, | Nasdaq: SRPT |
| 46 | SQZ Biotechnologies | The company’s Cell Squeeze technology addresses barriers to cell therapy development and implementation. | LGMD2E β-sarcoglycan, LGMD2D α-sarcoglycan | NYSE: SQZ |
| 47 | Vertex Pharmaceuticals | Vertex has acquired Exonics and has a partnership with CRISPR Therapeutics to develop a gene-editing platform for Duchenne’s muscular dystrophy (DMD) and myotonic dystrophy (DM1). | CRISPR/Cas9 | Nasdaq: VRTX |
| 48 | Verve Therapeutics | The company’s single-course gene editing programs focus on conditions with a genetically driven, life-long and severely elevated LDL-C such as familial hypercholesterolemia (FH). | VERVE-101/ABE-PCSK9, ANGPTL3 | Nasdaq: VERV |
| 49 | Vizgen | Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. | The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. | privately held |
| 50 | Voyager Therapeutics | Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. The company aims to develop therapies for neurological disorders and other diseases. | Specializes in developing next-generation AAV capsids for gene therapies. | Nasdaq: VYGR |
Filed Under: Cell & gene therapy
