Gene Therapy Market is projected to reach USD 17.2 billion by 2027 from USD 7.3 billion in 2022, at a CAGR of 18.6% during the forecast period. The lucrative growth is attributed to factors such as the high incidence of cancer and other target diseases, increasing product approvals, and the availability of funding for gene therapy research. On the other hand, the high cost of treatment and the availability of alternative treatment methods are expected to limit market growth to a certain extent.

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Key Market Players

Key players in the global Cell and gene therapy market include Biogen (US), Sarepta Therapeutics (US), Gilead Sciences, Inc. (US), Amgen, Inc. (US), Novartis AG (Switzerland), Orchard Therapeutics Plc (UK), Spark Therapeutics, Inc. (A Part Of ýF. Hoffmann-La Roche) (US), AGC Biologics (US), Anges, Inc. (Japan), Bluebird Bio, Inc. (US), Jazz Pharmaceuticals Plc (Ireland), Dynavax Technologies (US), Human Stem Cells Institute (Russia), Sibiono Genetech Co., Ltd. (China), Shanghai Sunway Biotech Co., Ltd. (China), Uniqure N.V. (Netherland), Gensight Biologics S.A. (France), Celgene Corporation (A Bristol-Myers Squibb Company) (US), Cellectis (France), Sangamo Therapeutics (US), Mustang Bio (US), AGTC (Applied Genetic Technologies Corporation) (US), and Poseida Therapeutics, Inc. (US).

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Gene Therapy Market Dynamics:

Drivers: Funding for gene therapy research

Gene therapy is one of the latest treatment methods in the healthcare industry. It has shown promising results in treating cancer and other genetic diseases. As a result, the focus on gene therapy research has increased over the last few years. In addition, many companies focus on growth opportunities in this market by launching new and advanced products.

For example:

  • In March 2022, the National Heart Lung and Blood Institute granted USD 12 million to an Indiana University School of Medicine researcher. The grant is for exploring major themes in a gene therapy approach to safer and potentially curative treatments for hemophilia.
  • In May 2019, Locana, Inc., a leading RNA-targeting gene therapy company, received USD 55 million in Series A financing led by ARCH Venture Partners, with participation from Temasek and Lightstone Ventures (all existing investors). The company will utilize this fund for its RNA-targeting gene therapy pipeline programs.

Opportunities: Strong product pipeline

Gene therapy is a promising revenue-generation area for pharmaceutical & biotechnology companies. Currently, most key players are focusing on new gene therapy products to capitalize the gene therapy market growth opportunities. Some of these products are in the pre-clinical stage, while others are in the clinical phase and are expected to secure approval in the coming years. In addition, companies operating in the gene therapy industry are focusing on various disease areas, including hemophilia A & B, Fabry disease, wet age-related macular degeneration, mucopolysaccharidosis type II, ornithine transcarbamylase (OTC) deficiency, achromatopsia, and multiple myeloma. As a result, the patient pool for gene therapies is expected to increase in the coming years.

Challenges: High treatment cost

Currently, the cost of gene therapies is very high and can reach up to USD 1 million for one patient, depending on the disease condition. Most gene therapy products in the US and EU are covered under reimbursement programs. However, it is difficult for people not covered by insurance to afford such high-cost treatments.

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Recent Developments:

  • In February 2022, Sarepta Therpeutics (US) announced a collaboration to use GenEdit’s NanoGalaxy platform and Sarepta’s gene editing technology to develop gene editing therapeutics to treat neuromuscular diseases.
  • In May 2022, Novartis AG (Switzerland) received FDA approval for KYMRIAH for adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

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