Insider Ranking

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.

Courtesy of Himanshu Sehgal and Shivani Chandra of PharmaShots*

05 Takeda

Total Revenue: $29.40B

Approved Therapy: Alofisel

Headquarters: Tokyo, Japan

An R&D-driven global biopharma company, Takeda is committed to discovering and delivering products across therapy areas. 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.

04 Celgene

Total Revenue: $46.16B

Approved Therapy: Breyanzi & Abecma

Headquarters: New York, United States

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 for the treatment of large B-cell lymphoma. Additionally, in collaboration with 2seventybio (a bluebird bio company), BMS also received approval for Abecma for the treatment of multiple myeloma.

03 Spark Therapeutics

Total Revenue: $49.27B

Approved Therapy: Luxturna

Headquarters: Basel, Switzerland

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 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.

02 Novartis

Total Revenue: $50.54B

Approved Therapy: Zolgensma & Kymriah

Headquarters: Basel, Switzerland

Novartis is a global biopharma company that discovers, develops, and delivers pharma products to treat unmet medical conditions in therapy areas. Kymriah is the company’s first cell therapy approved for treating acute lymphocytic leukemia that utilizes the patient’s T cells to fight and kill cancer cells. Zolgensma, for its part, is Novartis’ approved gene therapy for treating spinal muscular atrophy (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.

01 Merck

Total Revenue: $52B

Approved Therapy: Adstiladrin

Headquarters: New Jersey, United States

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 CalmetteGuérin unresponsive non-muscle invasive bladder cancer.

*Based on annual reports, SEC filings, press releases and company websites. Presented in mid-2023 by PharmaShots.