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

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