Cell and Gene Therapy Market: Introduction
- Cell and gene therapy has high potential to eliminate and prevent several chronic disorders as well as life-threatening disorders especially cancer, heart diseases, AIDS, cystic fibrosis, and age-related disorders. The therapy provides complete cure to patients affected with chronic disorders rather than ease of symptoms offered by other treatments.
- The gene therapy concept emerged in 1989. Since then, researchers have been attempting to leverage the potential of gene therapy for treatment of rare and genetic diseases as well as diseases for which complete cure is not available yet.
- The first gene therapy product ‘Gendicine’ was developed by Shenzhen SiBiono GeneTech Co. Ltd. in 2003. It was commercialized for the treatment of head and neck cancer in 2004. Since 2004, more than 30,000 patients in China have been treated with Gendicine. Gendicine has exhibited exemplary safety results. When gene therapy is combined with other treatments, such as chemotherapy and radiotherapy, the overall outcome outperforms other standard therapies used alone.
Key Drivers and Restraints of Global Cell and Gene Therapy Market
- Technological advancements in cell and gene therapy is one of the key factors projected to fuel the global cell and gene therapy market during the forecast period. Emerging technologies in cell and gene therapies, such as proprietary cell lines, gene vectors, cell expansion and separation systems, and single-use bioprocessing reactors, have become primary means by which single products have been transformed into a robust product portfolio. For example, Immunicum has offered three technological platforms i.e. gene editing, CAR-T cell expansion, and T-cell primers. These technologies allow the company to advance its series of immuno-oncology drug candidates. Immunicum has two technologies i.e. IMM-2 platform and IMM-3 platform undergoing preclinical studies for use in the treatment of different types of cancer. Immunicum is looking forward to developing an allogeneic dendritic cell in-vivo vaccine for use in the treatment of solid tumors.
- Leading players operating in the immuno-oncology field have formed around 40 partnership programs regarding high-value cell and gene therapies. Thus, development of various technological platforms across a wide range of disease indications is one of the key factors driving the global cell and gene therapy market.
- Cell and gene therapy is one of the most expensive treatments available in the world. High costs of cell and gene therapy and limited reimbursements are preventing patients from receiving treatments.
- For instance, Spark Therapeutics Inc.'s gene therapy product ‘Luxturna’ used for the treatment of retinal dystrophy is priced at US$ 850,000 per treatment. It is the world’s most expensive drug. Orchard Therapeutics’ gene therapy product ‘Strimvelis,’ which is used for the treatment of ADA-SCID is priced around US$ 648,000 per treatment.
Oncology Segment to Lead Global Cell and Gene Therapy Market
- Approval of new gene therapy products and growth potential of gene therapy have led to need for increase in the number of treatment centers in the U.S. as well as Europe. Market players, after the initial success of their gene therapies, are striving to increase the number of treatment centers to extend the access to a larger pool of patients requiring gene therapy.
- In the first year of approval of Yescarta, a drug by Gilead, i.e. in 2017, only 15 authorized cancer hospitals were operating in the U.S. By the end of December 2017, only five patients were treated with Yescarta, whereas the waiting list had more than 200 patients. In October 2018, Gilead planned to increase the number of gene therapy centers to 90. As of June 2018, around 68 treatment centers were operating in the U.S. Also, the company has launched the ‘Kite Connect’ program to provide information and assistance, including insurance benefits, throughout the Yescarta therapy process.
- A gene therapy product called Kymriah, which was approved in 2017 for use in the treatment of patients aged up to 25 years and affected with large B-cell lymphoma, has only 41 treatment centers in the U.S. This is limiting a significant number of patients in the country from receiving gene therapy as a treatment.
Key Players Operating in Global Cell and Gene Therapy Market
Manufacturers in the global cell and gene therapy market are increasingly investing in research and development of new and innovative techniques to manufacture advanced cell and gene therapies. These players are also focused on offering highly efficient and reliable products to provide better protection from fragility. The global cell and gene therapy market is less fragmented, with the presence of a small number of regional players. Market players focus on strategic partnerships, collaborations, mergers, acquisitions, and new product launches to gain higher market share. In December 2019, Merck signed a license agreement with Promega Corp. to provide the former’s foundational CRISPR intellectual property to the latter. As per the agreement, Promega would be using Merck’s CRISPR genome editing technology to develop research products and services, including those for drug development.
Leading companies operating in the global cell and gene therapy market are:
- Gilead Sciences, Inc.
- Amgen, Inc.
- Novartis AG
- Orchard Therapeutics Plc
- Spark Therapeutics, Inc. (A Part of Hoffmann-La Roche)
- Molmed S.P.A.
- Anges, Inc.
- Bluebird Bio, Inc.
- Human Stem Cells Institute (HSCI)
- SIBIONO Genetech Co., Ltd.
- Shanghai Sunway Biotech Co., Ltd
- Uniqure N.V.
- Gensight Biologics S.A.
- Celgene Corporation (A Bristol-Myers Squibb Company)
Global Cell and Gene Therapy Market: Research Scope
Global Cell and Gene Therapy Market, by Application
- Autoimmune Diseases
- Cardiovascular Disorders
- Central Nervous System
- Orthopedic Disorders
Global Cell and Gene Therapy Market, by Therapy
- Cell Therapy
- Human Embryonic Stem Cells
- Hematopoietic Stem Cells
- Mesenchymal Stem Cells
- Gene Therapy
- Non-viral Vectors
- Viral Vectors