The global Gene Therapy Market by Vectors (Non-viral(Oligonucleotides), Viral(Retroviral, Adeno-associated)), Indication (Cancer, Neurological, Hepatological Diseases, Duchenne Muscular Dystrophy), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2027", is projected to reach USD 17.2 billion by 2027 from USD 7.3 billion in 2022, at a CAGR of 18.6% between 2022 and 2027. Factors such as growing cases of cancer and neurological diseases, rising gene therapy product approvals, and increasing investment in research and development of gene therapies will drive the market growth. However, factors such as high cost associated with gene therapy are restraining the growth of this market.

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Browse in-depth TOC on "Gene Therapy Market”

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Key Market Players

The gene therapy market is consolidated, with three major players holding the dominant share of the market. In 2021, Biogen (US), Novartis AG (Switzerland), and Gilead Sciences, Inc. (US) were the leading players in the gene therapy market. Other major players in this market include Sarepta Therapeutics (US), Amgen, Inc. (US), Spark Therapeutics, Inc. (US), AGC Biologics (Italy), Orchard Therapeutics plc. (UK), Sibiono (China), Alnylam Pharmaceuticals, Inc. (US), Human Stem Cells Institute (Russia), AnGes, Inc. (Japan), Dynavax Technologies (US), Jazz Pharmaceuticals, Inc. (Ireland), and Akcea Therapeutics (US). The intensity of competitive rivalry in the gene therapy market is high.

Driver: 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 gene and cell therapy companies focus on growth opportunities in this market by launching new and advanced products.

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.

Opportunity: 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 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, gene and cell therapy 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.

Novartis AG (Switzerland) was the leading player in the gene therapy market. The company offers two gene therapy products—KYMRIAH and ZOLGENSMA. Globally, the company has strong distribution channels and a good brand image. As a result, KYMRIAH registered significant growth in 2018—KYMRIAH registered sales of USD 587 million in 2021, up 24% from 2020. ZOLGENSMA registered sales of USD 1,351 million in 2021, up 47% from 2020. Novartis has strong distribution channels in the US. As a result, in 2021, the company generated 32.6% of its total revenue from the US alone.

Biogen (US) is the second-leading player in the gene therapy market. However, the revenue of SPINRAZA is declining year on year. The decline in SPINRAZA revenue was primarily brought on by a decline in demand because of heightened competition in the US and Germany and a decline in pricing in the US and the RoW markets, which were only partially offset by an uptick in sales volumes in Latin America and some distributor markets.

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The non-viral vectors segment accounted for the largest share in the gene therapy market, by vector, during the forecast period.

Based on vectors, the gene therapy market is segmented into non-viral vectors and viral vectors. Non-viral vectors accounted for the larger market share in 2021. Less immunogenicity than viral vectors coupled with easier mass production of non-viral vectors are the major factors driving the growth of this segment.

The Neurological diseases segment accounted for the largest share of the indication segment in the gene therapy market in 2021.

Based on indication, the gene therapy market is segmented into neurological diseases, cancer, duchenne muscular dystrophy, hepatological diseases, and other indications. The neurological diseases segment accounted for the largest share of the gene therapy market in 2021. The largest share is attributed to growing burden of neurological diseases coupled with rising adoption of gene therapy for the treatment of neurological disorders.

The in vivo segment accounted for the larger share of the delivery method segment in the gene therapy market in 2021.

On the basis of delivery method, the gene therapy market is segmented into in vivo and ex vivo. In vivo segment accounted for the larger share in the gene therapy market as majority of the approved gene therapy products fall under in vivo delivery method.

North America is the largest region and Asia Pacific is the fastest-growing region of the gene therapy market in 2021.

Based on region, the gene therapy market is segmented into North America, Europe, Asia Pacific, and Rest of the World. In 2021, North America dominated the market, followed by Europe. The significant government and private investments to develop advanced gene therapy products, the growing adoption rate of these products, and the presence of key market players in the region are responsible for the large share of North America in the gene therapy market. The Asia Pacific market is projected to grow at the highest CAGR during the forecast period.

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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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