Top 20 Cell and Gene Therapy Companies Based on 2022 Total Revenue

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Standing tall and gradually paving its way through extensive research, Cell and Gene Therapy is a beacon of hope when treating rare and inherited diseases. Cell and gene therapy aims to prevent, treat, and potentially reduce the effects of the underlying cause of genetic diseases.
Propelled by constant innovatory winds, the Cell and Gene therapy market is anticipated to grow by 20 percent year-over-year through 2025. With a total revenue of $52B, Merck & Co. Was ranked the highest among the list of companies developing Cell & Gene therapy, followed by Novartis with a revenue of $50.54B and Spark Therapeutics with a revenue of $49.27B
PharmaShots brings an informative report on the Top 20 Cell and Gene Therapy Companies based on the total revenue generated in the year 2022

Total Revenue: $0.5M

Approved Cell and Gene Therapy: Collategene

Founded Year: 1999

Total Employees: ~140

Headquarters: Osaka, Japan

Market Cap: $162.8M

Stock Exchange: TYO

With focus areas extending to the discovery and development of biopharma products, AnGes is dedicated to developing genetic medicines and therapeutic vaccines for intractable or rare diseases. The company received its first approval from the Japanese government in 2019 to treat critical limb ischemia. AnGes and Mitsubishi Tanabe Pharma jointly developed Collategene. This gene therapy delivers a plasmid expressing hepatocyte growth factor (HGF) into the lower limb muscles causing an improved local blood flow and ulcer healing.

Total Revenue: $3.6M

Approved Cell and Gene Therapy: Skysona & Zynteglo

Founded Year: 1992

Total Employees: ~323

Headquarters: Massachusetts, United States

Market Cap: $397.9M

Stock Exchange: NASDAQ

Committed to pharmaceutical products' research, development, and commercialization, bluebird bio utilizes its lentiviral vector gene addition platform to develop gene therapies for treating severe genetic diseases. The company first received the US FDA approval for its gene therapy Zynteglo in Aug 2022 for treating adult and pediatric patients with ß-thalassemia. Zynteglo adds functional copies of the modified β-globin gene to the patient's hematopoietic stem cells (HSCs). Later in Sep 2022, the company also received approval from the US FDA for its gene therapy, Skysona, to treat boys aged between 4 and 17 with active cerebral adrenoleukodystrophy (CALD). Skysona functions by lowering the progression of neurologic dysfunction in CALD patients. In November 2021, bluebird bio spun off its Oncology business into a separate company named 2seventy Bio.

Total Revenue: $20M

Approved Cell and Gene Therapy: Libmeldy & Strimvelis

Founded Year: 2015

Total Employees: ~170

Headquarters: London, United Kingdom

Market Cap: $100.6M

Stock Exchange: NASDAQ

Orchard Therapeutics is a company that develops and commercializes gene therapies to treat the underlying cause of diseases. The company has two approved gene therapies, Libmeldy and Strimvelis. Libmeldy received marketing authorization from the EMA on Dec 2020 for the treatment of Metachromatic Leukodystrophy (MLD), whereas, Strimvelis obtained the EMA’s approval in May 2016 for the treatment of Adenosine Deaminase Deficiency. Libmeldy is a genetically modified autologous CD34+ cell-enriched population containing HSPC transduced ex vivo using a lentiviral vector expressing the human arylsulfatase A (ARSA) gene. Strimvelis, an autologous CD34+ enriched cell fraction, is composed of CD34+ cells that have been retrovirally transduced with a retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human hematopoietic stem/progenitor (CD34+) cells.

Total Revenue: $21M

Approved Cell and Gene Therapy: Carteyva (Relma-cel)

Founded Year: 2016

Total Employees: ~90

Headquarters: Shanghai, China

Market Cap: $165.05M

Stock Exchange: HKG

Marking its position in the Cell and Gene Therapy market, JW Therapeutics is a biotech company working on developing, manufacturing, and commercializing cell immunotherapy products. The company develops therapies for therapeutic areas, including hematologic malignancies, solid tumors, and autoimmune diseases. Its Carteyva (Relma-cel) received the NMPA's approval in Sep 2021 for treating large B-cell lymphoma. Carteyva is an autologous anti-CD19 CAR-T cell immunotherapy developed by JW Therapeutics using Juno Therapeutics' CAR-T cell process platform.

Total Revenue: $45M

Approved Cell and Gene Therapy: Apligraf & Novachor

Founded Year: 1985

Total Employees: ~1,030

Headquarters: Massachusetts, United States

Market Cap: $420.04M

Stock Exchange: NASDAQ

A regenerative medicine company, Organogenesis is a pharma company that develops, manufactures, and commercializes Advanced Wound Care and Surgical and Sports Medicines. Apligraf and Novachor are the two approved gene therapy developed by the company. Apligraf, approved in 1998, is a bioengineered living cell therapy containing keratinocytes and fibroblasts producing a wide range of cytokines and growth factors. Approved in the year 2021, Novachor is a fresh chorion membrane wound covering developed as a preservative of viable cells, growth factors, cytokines, and ECM proteins in native tissue.

Total Revenue: $110M

Approved Cell and Gene Therapy: Hemgenix

Founded Year: 1998

Total Employees: ~500

Headquarters: Amsterdam, Netherlands

Market Cap: $1.01B

Stock Exchange: NASDAQ

Delving into the Cell and Gene Therapies market, uniQure is a biopharma company that develops and commercializes gene therapies to treat rare diseases. Amongst the pipeline of gene therapy being evaluated, In Nov 2022, the company received approval for Hemgenix from the US FDA to treat Hemophilia B. Hemgenix is an adeno-associated virus vector serotype 5 (AAV5) based gene therapy designed to deliver a copy of a gene encoding Padua variant of human coagulation Factor IX (hFIX-Padua)

Total Revenue: $117M

Approved Cell and Gene Therapy: Carvykti

Founded Year: 2014

Total Employees: ~1,400

Headquarters: New Jersey, United States

Market Cap: $11.89B

Stock Exchange: NASDAQ

Legend Biotech is a multinational biotech company that develops, manufactures, and commercializes therapies for treating life-threatening diseases. It is a holding company that conducts operations through its subsidiaries, including its PCR subsidiaries. In Dec 2017, Legend Biotech entered into a collaboration agreement with Janssen Pharmaceutical to develop and commercialize Carvykti. The company’s gene therapy candidate, Carvykti, received the US FDA's approval in Mar 2022 for treating Multiple Myeloma, the NDA filed by Janssen. It also received approval from the EMA and MHLW in May and Sep 2022. Carvykti is a BCMA-directed genetically modified autologous T-cell immunotherapy that involves the reprogramming of the patient's T-cells with a transgene encoding a CAR that identifies and eliminates BCMA-expressing cells

Total Revenue: $164M

Approved Cell and Gene Therapy: MACI

Founded Year: 1987

Total Employees: ~300

Headquarters: Massachusetts, United States

Market Cap: $1.55B

Stock Exchange: NASDAQ

A biopharma company marking its way into the Cell and Gene Therapies Market, Vericel Corp. develops and commercializes gene therapies targeting unmet therapeutic needs. MACI is the company’s only approved product to date. The company received the US FDA’s approval for MACI in Dec 2016 for the repair therapy of cartilage defects in patients with knee problems. MACI is developed using the patient’s autologous cells expanded and placed into a bio-resorbable porcine-derived collagen membrane implanted over the area from which the defective or damaged tissue was removed

Total Revenue: $302M

Approved Cell and Gene Therapy: Temcell

Founded Year: 1975

Total Employees: ~800

Headquarters: Ashiya, Japan

Market Cap: $1.30B

Stock Exchange: TYO

Aimed toward the research, development, manufacturing, and marketing of biopharma and regenerative products, JCR Pharmaceuticals has a well-established position in the Cell and Gene Therapies Market. The company utilizes technologies for cell therapy, regenerative medicines, and gene therapy technology to develop drugs and drug discovery platforms. JCR’s allogeneic cell therapy product, Temcell, was approved by the MHLW in Feb 2016 for the treatment of acute graft versus host disease (aGVHD) in children and adults

Total Revenue: $530M

Approved Cell and Gene Therapy: Upstaza

Founded Year: 1998

Total Employees: ~1,400

Headquarters: New Jersey, United States

Market Cap: $4.33B

Stock Exchange: NASDAQ

PTC Therapeutics discovers, develops, and commercializes products, including gene therapies, for treating rare diseases. Upstaza is the company’s approved gene therapy. In Jul 2022, the EMA granted Upstaza marketing authorization to treat adults and children aged 18 years or above with severe aromatic L-amino acid decarboxylase (AADC). Upstaza contains a functional version of the AADC gene, eladocagene exuparvovec, within a modified virus.

Total Revenue: $1.9B

Approved Cell and Gene Therapy: StrataGraft

Founded Year: 1867

Total Employees: ~2,700

Headquarters: Dublin, Ireland

Market Cap: $82.98M

Stock Exchange: NYSE

Mallinckrodt is a biopharma company focused on the development, manufacturing, marketing, and commercialization of pharma products and therapies for therapy areas, including Autoimmune and Rare Diseases, with a specialty in Neurology, Rheumatology, Hepatology, Nephrology, Pulmonology, Ophthalmology, and Oncology. The company's cell therapy portfolio contains a US FDA-approved StrataGraft. StrataGraft was approved by the US FDA in Jun 2021 for treating adults with deep partial-thickness burns. Keratinocytes and dermal fibroblasts are the two human skin cells incorporated in the development of StrataGraft, which are grown together to form a bi-layered construct on the skin.

Total Revenue: $2.09B

Approved Cell and Gene Therapy: Roctavian

Founded Year: 1997

Total Employees: ~3,100

Headquarters: California, United States

Market Cap: $17.25B

Stock Exchange: NASDAQ

BioMarin is a global biopharma company that develops and commercializes targeted therapies for genetic disorders. The company has Roctavian as its approved gene therapy. On Aug 2022, BioMarin received conditional marketing authorization for Roctavian from the EMA for treating severe hemophilia A. Roctavin is developed using valoctocogene roxaparvovec as the active gene. The product carries and delivers a short and functional copy of the F8 gene to the liver cells by using AAV5, an adeno-associated virus.

Total Revenue: $10.53B

Approved Cell and Gene Therapy: Delytact

Founded Year: 2005

Total Employees: ~16,450

Headquarters: Tokyo, Japan

Market Cap: $68.27B

Stock Exchange: TYO

Daiichi Sankyo discovers and develops products for therapy areas, including Oncology and Rare Diseases. Delytact is the company's approved gene therapy. In Jun 2021, the MHLW approved Daiichi to manufacture and market Delytact across Japan to treat patients with malignant glioma. Delytact is a genetically engineered oncolytic herpes simplex virus type 1 (HSV-1). It has triple mutation within the viral genome that causes augmented and selective replication in cancer cells and enhances induction of antitumor immune response.

Total Revenue: $26.32B

Approved Cell and Gene Therapy: Imygic

Founded Year: 1980

Total Employees: ~25,200

Headquarters: California, United States

Market Cap: $119.38B

Stock Exchange: NASDAQ

Amgen is an American biotech company that discovers, develops, and manufactures products targeting diseases with limited treatment options. The company has marked its spot in the cell and gene market through its approved gene therapy, Imlygic. Amgen received the US FDA’s approval for Imlygic in Oct 2015 and the EMA's approval Dec 2015 for treating unresectable cutaneous, subcutaneous, and nodal lesions in patients with recurrent melanoma after the initial surgery. Imlygic is a genetically modified herpes simplex virus type 1, which, when injected inside the tumor, replicates and produces an immunostimulatory protein, GM-CSF.

Total Revenue: $27.28B

Approved Cell and Gene Therapy: Tecartus & Yescarta

Founded Year: 1987

Total Employees: ~17,000

Headquarters: California, United States

Market Cap: $98.35B

Stock Exchange: NASDAQ

An American Biotech company, Kite Pharma, develops immunotherapy products for the treatment of cancers. Kite became an integral part of Gilead Sciences following its acquisition in 2017 by Gilead Sciences. The company primarily focuses on developing genetically engineered CAR T cell therapies. Tecartus & Yescarta are two approved CAR-T cell therapies developed by Kite Pharma. Tecartus was approved by the EMA (Dec 2020) and US FDA (Oct 2021), whereas, Yescarta was approved by the EMA (Aug 2018), TGA (Feb 2020), MHLW (Jan 2021; filed by Daiichi Sankyo), US FDA (Apr 2022), and Health Canada (Mar 2023) for the treatment of Mantle cell lymphoma.

Total Revenue: $29.40B

Approved Cell and Gene Therapy: Alofisel

Founded Year: 1781

Total Employees: ~50,000

Headquarters: Tokyo, Japan

Market Cap: $51.24B

Stock Exchange: TYO

An R&D-driven global biopharma company, Takeda is committed to discovering and delivering products across therapy areas, including Oncology, Metabolic Disorders, Gastroenterology (GI), Neurology, and Immunology. Takeda's cell and gene therapy segment includes Alofisel. The MHLW approved Takeda to manufacture and commercialize Alofisel across Japan to treat patients with non-active or mildly active Luminal Crohn's Disease. The suspension of allogeneic develops Alofisel expanded adipose-derived stem cells (eASC) to treat complex perianal fistulas in patients with Luminal Crohn's Disease.

Total Revenue: $46.16B

Approved Cell and Gene Therapy: Breyanzi & Abecma

Founded Year: 1887

Total Employees: ~34,300

Headquarters: New York, United States

Market Cap: $138.32B

Stock Exchange: NYSE

Bristol Myers Squibb (BMS) is a global biopharma company dedicated to developing and commercializing products targeting therapy areas, including Oncology, Cardiovascular, and Immunological Diseases. BMS's subsidiary, Celgene Corporation, is a pharmaceutical company primarily focusing on developing cancer treatment immunotherapies. Under its immunology cell therapy, Celgene has two approved therapies, Breyanzi and Abecma. Breyanzi was approved by the MHLW (Mar 2021), EMA (Apr 2022), and the US FDA (Jun 2022) for the treatment of large B-cell lymphoma. Breyanzi is a CAR T cell therapy that targets CD19 expressed on the Cell during normal B-cell development. Additionally, in collaboration with 2seventybio (a bluebird bio company), BMS also received approval for Abecma from the US FDA (Mar 2021), Health Canada (May 2021), and the EMA (Aug 2021) for the treatment of multiple myeloma. Abecma is a BCMA-directed CAR T immunotherapy.

Total Revenue: $49.27B

Approved Cell and Gene Therapy: Luxturna

Founded Year: 1896

Total Employees: ~1,03,600

Headquarters: Basel, Switzerland

Market Cap: $308.07B

Stock Exchange: SWX

Roche is a multination biopharma company that develops and commercializes products under therapy areas, including Oncology, Cardiovascular, and Immunological Diseases. Spark Therapeutics' subsidiary discovers and delivers gene therapies for treating genetic disorders. Spark's Luxturna was approved by the US FDA (Dec 2017), EMA (Nov 2018), TGA (Aug 2020), and Health Canada (Oct 2020) as a gene therapy for the treatment of Leber's congenital amaurosis. Luxturna is developed using the active substance vortigene neparvovec. Earlier in Jan 2018, Spark Therapeutics entered into a licensing agreement with Novartis to develop and commercialize Luxturna in areas outside the US.

Total Revenue: $50.54B

Approved Cell and Gene Therapy: Zolgensma & Kymriah

Founded Year: 1996

Total Employees: ~102,000

Headquarters: Basel, Switzerland

Market Cap: $229.11B

Stock Exchange: SWX

Novartis is a global biopharma company that discovers, develops, and delivers pharma products to treat unmet medical conditions in therapy areas, including Oncology, Hepatology, Immunology, Dermatology, Musculoskeletal Diseases, Neurosciences, and Ophthalmology. Kymriah is the company's first cell therapy approved for treating acute lymphocytic leukemia. Kymriah was approved by the EMA (Aug 2018), Health Canada (Sep 2018), TGA (Dec 2018), MHLW (Mar 2019), and the US FDA (May 2022). Kymriah is a T-cell therapy that utilizes the patient's T cells to fight and kill cancer cells. Zolgensma is Novartis' approved gene therapy for treating spinal muscular atrophy (SMA). It has received approval from the US FDA (May 2019), MHLW (Mar 2020), EMA (May 2020), and Health Canada (Dec 2020) for the treatment of SMA. Zolgensma replaced the missing or defective SMN1 gene with a new copy of the gene in the patient's Cell, thereby inhibiting disease progression.

Total Revenue: $52B

Approved Cell and Gene Therapy: Adstiladrin

Founded Year: 1891

Total Employees: ~69,000

Headquarters: New Jersey, United States

Market Cap: $293.05B

Stock Exchange: NYSE

With focus areas extending to Cardiovascular, Diabetes, Oncology, Neurology, and Immunology, Merck is a multinational biopharma company that has also marked a significant spot amongst the companies developing Cell and Gene Therapies. Adstiladrin is the only medication the company has approved for cell and gene therapy. In Dec 2022, the US FDA approved Merck's Adstiladrin for treating high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer. Adstiladrin is a non-replicating adenoviral vector-based gene therapy that delivers a copy of the gene encoding a human interferon-alfa 2b (IFNα2b) to the bladder urothelium.

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