Shots:
- Cell and gene therapies provide a promising opportunity to enhance care for unmet healthcare needs. Biopharma companies are actively exploring these therapies across multiple indications to address healthcare gaps in rare and chronic diseases
- In 2023, the cell and gene therapy market was valued at $18.2B and is projected to grow to $117.46B by 2034, with a CAGR of 18.6%
- PharmaShots presents a concise report on the top 20 cell and gene therapy companies of 2024. In ranking companies, greater emphasis is placed on those with a higher number of late-stage products, even if the total number of pipeline candidates is the same
| Rank | Companies | No. of Products in Pipeline |
| 1 | Cellectis | 7 |
| 2 | Bayer | 6 |
| 3 | MeiraGTx (MEIRAGTX HOLDINGS PLC) | 6 |
| 4 | Rocket Pharmaceuticals | 5 |
| 5 | 4D Molecular Therapeutics | 5 |
| 6 | CRISPR Therapeutics | 5 |
| 7 | Roche | 5 |
| 8 | Ultragenyx | 4 |
| 9 | Abeona Therapeutics | 4 |
| 10 | Bristol-Myers Squibb | 4 |
| 11 | Regeneron | 4 |
| 12 | uniQure | 4 |
| 13 | Krystal Biotech, Inc. | 4 |
| 14 | Beam Therapeutics | 4 |
| 15 | Allogene Therapeutics | 4 |
| 16 | Gilead Sciences, Inc. ( KITE) | 4 |
| 17 | Autolus Therapeutics | 4 |
| 18 | Intellia Therapeutics | 3 |
| 19 | Sarepta Therapeutics | 3 |
| 20 | Arcellx | 3 |
Note: Columns 1 and 2 represent the Rank and Companies, while Columns 3 showcase the total pipeline products
20. Arcellx
Pipeline Candidates: Anito-cel, ACLX-001, ACLX-002
Founded Year: 1980
Total Employees: 1314
Headquarters: Massachusetts, United Nations
Market Cap: $4.79B
Stock Exchange: NASDAQ
- Arcellx is a clinical-stage biotechnology company focused on developing innovative immunotherapies for patients with cancer and other unmet medical needs
- Its pipeline features three key cell therapy candidates: Anito-cel, ACLX-001, and ACLX-002. Anito-cel (P-III) and ACLX-001 (P-I) are being developed for the treatment of relapsed or refractory multiple myeloma, while ACLX-002 (P-I) is aimed at treating relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome
- In Nov’23, Kite (Gilead) and Arcellx Reported the Expansion of their Collaboration Agreement for CART-DdBCMA in Multiple Myeloma
19. Sarepta Therapeutics
Pipeline Candidates: SRP-9003, SRP-9004, SRP-6004
Founded Year: 1980
Total Employees: 1314
Headquarters: Massachusetts, United Nations
Market Cap: $11.53B
Stock Exchange: NASDAQ
- Sarepta is a commercial-stage biotechnology company focused on discovering and developing RNA-targeted therapies, gene therapies, and gene therapy modalities for rare diseases
- The company’s clinical pipeline includes SRP-9003, SRP-9004, and SRP-6004, which are being developed to treat limb-girdle muscular dystrophies. Currently, Sarepta has one approved gene therapy and three approved RNA-based therapies
- In Jun’23, Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec- rokl) Received the US FDA’s Accelerated Approval for the Treatment of Duchenne Muscular Dystrophy.
18. Intellia Therapeutics
Pipeline Candidates: Nexiguran ziclumeran, NTLA-2002, NTLA-3001
Founded Year: 2014
Total Employees: 526
Headquarters: Massachusetts, United Nations
Market Cap: $1.5B
Stock Exchange: NASDAQ
- Intellia leverages CRISPR-based gene-editing technologies to develop novel in-vivo and ex-vivo curative gene and cell therapies aimed at treating genetic disorders, autoimmune diseases, and various cancers
- Intellia’s NTLA-2001, currently in P-III development, is being advanced in collaboration with Regeneron for the treatment of transthyretin amyloidosis (ATTR). NTLA-2002 and NTLA-3001 are wholly owned CRISPR candidates, with NTLA-2002 in P-III and NTLA-3001 in P-I clinical trials
- In Oct’23, Intellia’s NTLA-2002 received PRIME designation from EMA.
17. Autolus Therapeutics
Pipeline Candidates: AUTO1/22, AUTO-4, AUTO6-NG, AUTO-8
Founded Year: 2014
Total Employees: 463
Headquarters: London, United Kingdom
Market Cap: $825.3M
Stock Exchange: NASDAQ
- Autolus is a UCL spinout company focused on developing autologous CAR-T cell therapies for cancer and autoimmune diseases
- With four cell therapies in P-I, Autolus aims to target T-cell lymphoma, solid tumors, B-cell malignancies, and multiple myeloma. BioNTech has the option to co-fund and co-commercialize AUTO1/22 and AUTO6-NG
- In Nov’23, Autolus submitted BLA to the US FDA for Obe-cel (obecabtagene autoleucel) to treat Relapsed/refractory (r/r) Adult B-cell Acute Lymphoblastic Leukemia.
16. Kite (Gilead Sciences, Inc.)
Pipeline Candidates: KITE-363, KITE-753, KITE-197, Anitocabtagene autoleucel
Founded Year: 2009
Total Employees: 18000
Headquarters: California, United States
Market Cap: $113.7B
Stock Exchange: NASDAQ
- Kite develops cancer immunotherapies with a primary focus on genetically engineered autologous CAR-T cell therapies
- Kite’s pipeline includes four cell therapies. Anitocabtagene autoleucel is currently being investigated in P-II, while the other three are in P-I for the treatment of B-cell lymphoma and multiple myeloma
- In Sept’23, Kite Reported P-II Study (ALYCANTE) Results of Yescarta (axicabtagene ciloleucel) for Relapsed/Refractory Large B-Cell Lymphoma
15. Allogene Therapeutics
Pipeline Candidates: Cema-cel, ALLO-715, ALLO-605, ALLO-316
Founded Year: 2017
Total Employees: 232
Headquarters: California, United States
Market Cap: $459.7B
Stock Exchange: NASDAQ
- Allogene Therapeutics is a clinical-stage biotech firm, focuses on developing allogeneic chimeric antigen receptor T-cell (AlloCAR-T) products to treat solid tumors, hematologic malignancies, and autoimmune diseases
- The clinical pipeline includes four AlloCAR-T products: Cema-cel, ALLO-715, ALLO-605, and ALLO-316. ALLO-715, ALLO-605, and ALLO-316 are licensed from Cellectis
- In Feb’23, Allogene Therapeutics presented data on their next-generation AlloCAR-T platform technology called Dagger.
14. Beam Therapeutics
Pipeline Candidates: BEAM-201, BEAM-101, BEAM-302, BEAM-301
Founded Year: 2017
Total Employees: 436
Headquarters: Massachusetts, United Nations
Market Cap: $2.18B
Stock Exchange: NASDAQ
- Beam Therapeutics leverages a suite of gene editing and delivery technologies to develop long-term cures for patients with serious medical conditions
- The genetic medicine pipeline includes BEAM-201, BEAM-101, BEAM-302, and BEAM-301, which are treatments for T-cell leukemia, AATD (Alpha-1 Antitrypsin Deficiency), sickle cell disease, and glycogen storage disorder type 1a, respectively
- In Oct’23, Beam and Lilly entered an agreement giving Lilly, Beam’s opt-in rights to Verve’s Base editing program for cardiovascular disease
13. Krystal Biotech, Inc.
Pipeline Candidates: KB-105, KB-407, KB-408, KB-707
Founded Year: 2016
Total Employees: 229
Headquarters: Pennsylvania, United States
Market Cap: $5.49B
Stock Exchange: NASDAQ
- Krystal is a commercial-stage gene therapy biotech company focused on discovering, developing, and marketing genetic medicines for the treatment of serious medical conditions
- Krystal’s VYJUVEK is a gene therapy indicated for the treatment of dystrophic epidermolysis bullosa. Additionally, the company has four clinical therapies under investigation in P-I/II for indications in dermatology, oncology, and respiratory conditions
- In Jul’23, Krystal Biotech Reported the First Patient Dosing of KB407 in the P-I Clinical Trial for the Treatment of Cystic Fibrosis.
12. UniQure
Pipeline Candidates: AMT-130, AMT‑191, AMT-162, AMT-260
Founded Year: 1998
Total Employees: 480
Headquarters: Amsterdam, Netherlands
Market Cap: $280.34M
Stock Exchange: NASDAQ
- UniQure advances gene therapy technologies using its AAV technology platform to address gaps in AAV gene therapy development
- Hemgenix, UniQure’s approved gene therapy for hemophilia, is complemented by its pipeline of clinical candidates, all in P-I/II stages. AMT-130 and AMT-162 have received Fast Track and Orphan Drug designations, while AMT-191 has received Orphan Drug designation from the US FDA
- In Feb’23, UniQure and CSL Published P-III Study (HOPE-B) Results of Hemgenix (etranacogene dezaparvovec-drlb) for Hemophilia in NEJM.
11. Regeneron
Pipeline Candidates: 27-T51, DB-OTO, Nexiguran ziclumeran (NTLA-2001), REGV131-LNP1265
Founded Year: 1988
Total Employees: 13,450
Headquarters: New York, United States
Market Cap: $81.30M
Stock Exchange: NASDAQ
- Regeneron is a biotech company focused on developing and commercializing therapies for serious and unmet health conditions. The company specializes in antibodies and genetic medicines
- The pipeline includes four clinical-stage cell and gene therapies: 27-T51, DB-OTO, Nexiguran Ziclumeran (NTLA-2001), and REGV131-LNP1265
- In Oct’23, Intellia and Regeneron announced the expansion of their research collaboration for the development of CRISPR-based therapies to treat neurological and muscular diseases.
10. Bristol-Myers Squibb
Pipeline Candidates: GPRC5D CAR T, BMS-986353, CD33 NKE, BMS-986453
Founded Year: 1887
Total Employees: 34,100
Headquarters: New Jersey, United States
Market Cap: $120.48B
Stock Exchange: NYSE
- Bristol-Myers Squibb (BMS) is a global biopharma company that discovers, develops, and delivers innovative therapies to address unmet healthcare needs. The company focuses on oncology, hematology, immunology, cardiovascular, and neuroscience
- The cell and gene therapy pipeline of BMS includes GPRC5D CAR T, BMS-986353, CD33 NKE, and BMS-986453, targeting conditions such as multiple myeloma and acute myeloid leukemia
- In Dec’23, BMS’ Abecma CAR T therapy was approved in Japan for relapsed or refractory multiple myeloma patients.
9. Abeona Therapeutics
Pipeline Candidates: TSHA-118, TSHA-102, UX-111, pz-cel
Founded Year: 1974
Total Employees: 84
Headquarters: Ohio, United States
Market Cap: $276.34M
Stock Exchange: NASDAQ
- Abeona Therapeutics is a clinical-stage biopharma company developing cell and gene therapies for rare conditions such as Recessive Dystrophic Epidermolysis Bullosa (RDEB), Stargardt Disease, and others
- Abeona’s pipeline includes three cell and gene therapy candidates: TSHA-118 for Infantile Batten Disease (CLN1 Disease), TSHA-102 for Rett Syndrome, UX-111 for Sanfilippo Syndrome Type A, and pz-cel for RDEB
- In Nov’23, the US FDA granted BLA to Abeona Therapeutics’ Pz-cel a gene therapy in development to treat RDEB.
8. Ultragenyx
Pipeline Candidates: UX-111, DTX-401, UX-701, DTX-301
Founded Year: 2010
Total Employees: 1,276
Headquarters: California, United States
Market Cap: $4.43B
Stock Exchange: NASDAQ
- Ultragenyx Pharmaceutical develops treatments for rare genetic diseases, focusing on biologics, small molecules, gene therapies, and ASO/mRNA approaches for bone, endocrine, metabolic, muscle, and CNS conditions
- The gene therapy pipeline at Ultragenyx includes UX-111, DTX-401, UX-701, and DTX-301, with three drug candidates currently in P-III trials
- In Jul’23, Ultragenyx reported the initiation of the dosing in the second cohort of the pivotal P-I/II/III Cyprus2+ trial assessing UX701 gene therapy to treat Wilson Disease.
7. Roche
Pipeline Candidates: RG-6540, RG-6357, RG-6501, RG-6538, RG-6182
Founded Year: 1896
Total Employees: 100,000
Headquarters: Basel, Switzerland
Market Cap: $265.47B
Stock Exchange: SWX
- Roche is a global healthcare company focused on the discovery, development, and commercialization of therapies and diagnostic products. The company operates in two main business segments: Pharmaceuticals and Diagnostics
- Roche’s pipeline includes five cell and gene therapy products: RG-6540, RG-6357, RG-6501, RG-6538, and RG-6182. These products are being developed in P-I and P-II studies for various indications, including hematologic tumors, hemophilia A, geographic atrophy, multiple myeloma, and multiple sclerosis
- In Oct’23, SpliceBio collaborates with Spark Therapeutics (subsidiary of Roche Holding AG) on gene therapy for inherited retinal disease.
6. CRISPR Therapeutics
Pipeline Candidates: CTX-112, CTX-131, CTX-310, CTX-320, CTX-211
Founded Year: 2013
Total Employees: 407
Headquarters: Zug, Switzerland
Market Cap: $4.21B
Stock Exchange: NASDAQ
- CRISPR Therapeutics is a gene-editing company focused on creating innovative, gene-based treatments for various diseases using its proprietary CRISPR/Cas9 platform
- Under its pipeline, CRISPR Therapeutics is developing five clinical drugs: CTX-112, CTX-131, CTX-310, CTX-320, and CTX-211. These drugs target immuno-oncology, autoimmune conditions, cardiovascular diseases, and regenerative medicine
- In Dec’23, the US FDA approved Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) for the treatment of sickle cell disease.
5. 4D Molecular Therapeutics (4DMT)
Pipeline Candidates: 4D-150, 4D-125, 4D-110, 4D-710, 4D-310
Founded Year: 2013
Total Employees: 147
Headquarters: California, United States
Market Cap: $377.88M
Stock Exchange: NASDAQ
- 4DMT is a clinical-stage genetic medicine company focused on developing therapies for ophthalmology and pulmonology, utilizing its proprietary therapeutic vector evolution platform and a robust product design and development engine
- 4DMT pipeline includes five clinical drug candidates: 4D-150, 4D-125, 4D-110, 4D-710, and 4D-310. The candidates 4D-150, 4D-125, and 4D-110 are based on the R100 intravitreal vector delivery system, while 4D-710 and 4D-310 utilize A101 aerosol and C102 IV delivery methods, respectively
- In Oct’23, EMA granted PRIME Designation to 4DMT’s 4D-150 for the treatment of wet AMD.
4. Rocket Pharmaceuticals
Pipeline Candidates: LV-RP-L301, LV-RP-L102, RP-A501, RP-A601, RP-L201
Founded Year: 1999
Total Employees: 268
Headquarters: New Jersey, United States
Market Cap: $1.29B
Stock Exchange: NASDAQ
- Rocket Pharmaceuticals, a late-stage biotech company, develops genetic therapies using AAV and LV platforms to address complex and rare disorders
- The company’s gene therapy pipeline includes LV-RP-L301, LV-RP-L102, RP-A501, RP-A601, and RP-L201
- In Oct’23, the US FDA accepted Rocket Pharmaceuticals’ BLA with priority review for RP-L201 (marnetegragene autotemcel) to treat Severe Leukocyte Adhesion Deficiency-I (LAD-I).
3. MeiraGTx (MeiraGTx Holdings PLC)
Pipeline Candidates: AAV-AQP1, AAV-GAD, Botaretigene sparoparvovec, AAV-RPE65, AAV-CNGB3, AAV-CNGA3
Founded Year: 2015
Total Employees: 419
Headquarters: New York, United States
Market Cap: $475.05M
Stock Exchange: NASDAQ
- MeiraGTx, a clinical-stage subsidiary of MeiraGTx Holdings, is focused on developing gene therapies through a robust pipeline of late-stage clinical programs using its proprietary platform
- The company’s gene therapy pipeline includes AAV-AQP1, AAV-GAD, botaretigene sparoparvovec, AAV-RPE65, AAV-CNGB3, and AAV-CNGA3
- In Dec’23, MeiraGTx entered into an asset purchase agreement with Janssen Pharmaceuticals for bota-vec for up to $415M.
2. Bayer
Pipeline Candidates: AB-1002, AB-1005, Bemdaneprocel, ACTUS-101, AB-1001, AB-1003
Founded Year: 1863
Total Employees: 99,723
Headquarters: Leverkusen, Germany
Market Cap: $20.86B
Stock Exchange: ETR
- Bayer is a global healthcare and nutrition company, with business segments in Crop science, Pharmaceuticals, and Consumer health. The company focuses on cardiology, gynecology, diabetes, oncology, and ophthalmology
- Bayer’s gene therapy pipeline includes AB-1002, AB-1005, bemdaneprocel, ACTUS-101, AB-1001, and AB-1003. AB-1002, AB-1005, ACTUS-101, AB-1001, and AB-1003 originated from AskBio, while bemdaneprocel is from BlueRock
- In Oct’23, Bayer invested $250M in a new cell therapy production plant in Berkeley, California, USA to support BlueRock Therapeutics’ bemdaneprocel (BRT-DA01)
1. Cellectis
Pipeline Candidates: UCART-22, UCART-123, UCART20x22, Cemacabtagene ansegedleucel, ALLO-715, ALLO-605, ALLO-316
Founded Year: 2000
Total Employees: 256
Headquarters: Paris, France
Market Cap: $132.53M
Stock Exchange: NASDAQ
- Cellectis is a clinical-stage biotech company developing cell and gene therapies across various indications using its TALEN and PulseAgile technologies
- Cellectis currently has seven products in its gene therapy pipeline: UCART-22, UCART-123, UCART20x22, cemacabtagene ansegedleucel, ALLO-715, ALLO-605, and ALLO-316. Cemacabtagene ansegedleucel is licensed to Servier and Allogene, while ALLO-715, ALLO-605, and ALLO-316 are licensed to Allogene
- In Nov’23, Cellectis entered into a strategic collaboration and investment agreement with AstraZeneca to accelerate the development of up to 10 novel cell & gene therapy candidates
Sources:
- Annual reports
- SEC Filings
- Press releases
- Company Websites
Related Post: Top 20 Cell and Gene Therapy Companies Based on 2022 Total Revenue
