Top 20 Cell and Gene Therapy Companies of 2024

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: