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50 leading cell and gene therapy companies

By Brian Buntz | July 26, 2022

Pharma50: 50 Leading Cell and gene therapy companies

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
Tagged With: Abeona Therapeutics, Adaptimmune Therapeutics, Adverum Biotechnologies, Alnylam, Arrowhead Pharmaceuticals, Aspen Neuroscience, Astellas Gene Therapies, Atara Biotherapeutics, Atherys, Autolus Therapeutics, AVROBIO, Bit Bio, Bluebird Bio, Bristol-Myers Squibb, Cabaletta Bio, Capricor Therapeutics, Caribou Biosciences, cell and gene therapy, cell and gene therapy companies, Cellectis, Cellular Biomedicine Group, Celularity, CRISPR Therapeutics, Deep Genomics, Editas Medicine, eGenesis, Freeline Therapeutics, Gamida Cell, Generation Bio, Graphite Bio, Intellia Therapeutics, Krystal Biotech, MaxCyte, MeiraGTx, Mesoblast, Metagenomi, Mission Bio, NexImmune, NextCure, Novartis AG, Passage Bio, Pluristem Therapeutics, Regenxbio Inc., Roche Holding AG, Sangamo Therapeutics, Sarepta Therapeutics Inc., SQZ Biotechnologies, Vertex Pharmaceuticals, Verve Therapeutics, Vizgen, Voyager Therapeutics
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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