Gene Therapy Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

The Gene Therapy Market is Segmented by Indication (Cancer, Metabolic Disorders, Eye Disorders, Spinal Muscular Atrophy, and Other Indications), Technology (Adeno Virus Vector, Adeno-associated Virus Vector, Lentiviral Vector, Retroviral Vector, Herpes Virus Vector, and Other Technologies), and Geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The report offers the value (in USD million) for the above segments.

Market Snapshot

Gene Therapy Market Size
Study Period: 2019-2027
Base Year: 2021
Fastest Growing Market: Asia Pacific
Largest Market: North America
CAGR: 25.71 %

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Market Overview

The gene therapy market is projected to register a CAGR of 25.71% during the forecast period (2022-2027).

The COVID-19 pandemic had a positive effect on the gene therapy market. Gene and cell therapy technology were extensively used in the development of vaccines used to treat COVID-19. For example, in January 2021, vaccine candidates developed by Mass General Brigham were made by utilizing gene-therapy technology that elicited strong immune responses in animal models for the treatment of COVID-19. The vaccine candidate was named AAVCOVID, and the researchers received a USD 2.1 million grant to further develop the technology from the Bill & Melinda Gates Foundation. The vaccine that was developed was far more convenient than the ones sold on the market, as it had a single dose and could be stored at room temperature. Also, as per a January 2021 published article titled "Emerging Patent Landscape for Gene Therapy as a Potential Cure for COVID-19", the treatment of coronavirus from the perspective of RNA interference-based gene therapy offered a more direct approach to combating viral genes and has a promising future. Thus, the COVID-19 pandemic led to an increased demand for gene-based therapy for COVID-19.

The major factors propelling the market's growth include technological advancements, rising investments in research and development, and the growing prevalence of target diseases.

Investment in research and development (R&D) activities are also expected to have a significant effect on the market. Several companies are aiming to build a gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities and enhancing those capabilities through strategic collaborations, expansion of R&D activities, as well as potential licensing, merger, and acquisition activities. For instance, in January 2022, the Cystic Fibrosis Foundation reported investing in SalioGen Therapeutics to support the company's preclinical research into novel gene therapy for cystic fibrosis. SalioGen's Gene Coding approach is designed to turn on, turn off, or modify the function of any gene in the genome. In March 2021, ElevateBio, one of the leading biotech companies focused on gene-based therapies, raised USD 525 million to advance its cell and gene therapy technologies.

However, the lack of standard regulations and the high price of products are the factors hindering the gene therapy market's growth.

Scope of the Report

As per the scope of this report, gene therapy is an advanced medical treatment that involves the transfer of a normal or healthy gene to replace a defective gene in a cell. It can be used to cure various chronic and genetic disorders in human beings, for which no final cure has been developed. The market is segmented by Indication (Cancer, Metabolic Disorders, Eye Disorders, Spinal Muscular Atrophy, and Other Indications), Technology (Adeno Virus Vector, Adeno-associated Virus Vector, Lentiviral Vector, Retroviral Vector, Herpes Virus Vector, and Other Technologies), and Geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The market report also covers the estimated market sizes and trends for 17 countries across major regions globally. The report offers the value (in USD million) for the above segments.

By Indication
Metabolic Disorders
Eye Disorders
Spinal Muscular Atrophy
Other Indications
By Technology
Adeno Virus Vector
Adeno-associated Virus Vector
Lentiviral Vector
Retroviral Vector
Herpes Virus Vector
Other Technologies
North America
United States
United Kingdom
Rest of Europe
South Korea
Rest of Asia-Pacific
Middle-East and Africa
South Africa
Rest of Middle-East and Africa
South America
Rest of South America

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Key Market Trends

Cancer is Expected to Hold Significant Market Share in the Indication Segment over the Forecast Period.

The factors boosting segment growth are the increasing burden of cancer disorder, the growing focus on research to develop an effective treatment for cancer, and rising investments pertaining to cancer research.

For instance, according to the American Cancer Society's 'Cancer Facts & Figures 2022', around 1,918,030 new cancer cases and 609,360 deaths due to cancers are estimated to be reported in the United States in the year 2022. In the treatment of cancer, various gene therapy strategies are currently employed. These include anti-angiogenic gene therapy, pro-drug activating suicide gene therapy, gene therapy-based immune modulation, oncolytic virotherapy, correction/compensation of gene defects, antisense, genetic manipulation of apoptotic and tumor invasion pathways, and RNAi strategies. The cancer types, such as brain, lung, breast, pancreatic, liver, colorectal, prostate, bladder, head and neck, skin, ovarian, and renal cancer, have been the target of these therapies.

A growing focus on developing effective therapies for cancer is also contributing to the market growth. For instance, in March 2021, the United States Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after at least four prior lines (different types) of therapy. Abecma is the first cell-based gene therapy approved by the Food and Drug Administration for the treatment of multiple myeloma.

Many key players in the market are focused on adopting strategies, such as mergers and acquisitions, on enhancing their product portfolio, which in turn, is expected to propel the market growth over the forecast period. For instance, in June 2021, CyGenica secured funding from SOSV Investments LLC, a venture capital company, to accelerate cancer and rare genetic disease therapy.

Thus, such developments are significantly contricbuting to the growth of the overall market.

Estimated Number of Cancer Cases and Deaths, by Sex, United States, 2022

North America Dominates the Market and is Expected to do Same over the Forecast Period.

Within North America, the United States has held a major share of the market. The major factors fueling the market growth are the increasing number of investments by the government, advancements in research and development about gene therapy, and the growing prevalence of target diseases.

For instance, as per the United States Center for Disease Control and Prevention article on spinal muscular atrophy (SMA) updated in December 2021, SMA is a genetic disorder that affects around 1 in every 10,000 people. Therefore, it is one of the most common rare diseases. The numbers indicate the growing demand for gene therapy in the United States. In May 2022, the Food and Drug Administration approved the drug, Evrysdi for babies under two months old with spinal muscular atrophy. Evrysdi is an SMN-enhancing therapy that works by targeting the SMN2 gene. Such product approvals in the region are adding to the growth of the market.

Several initiatives are being launched about gene therapy. For instance, as per an October 2021 update by the National Institutes of Health, the National Institutes of Health, United States Food and Drug Administration, 10 pharmaceutical companies, and 5 non-profit organizations have partnered to accelerate the development of gene therapies for the 30 million Americans who suffer from a rare disease. Such initiatives are anticipated to increase the demand for gene therapy, thereby contributing to market growth.

Thus, due to the above-mentioned developments, the market is expected to witness significant growth over the forecast period.

Gene Therapy Market Analysis

Competitive Landscape

The gene therapy market is highly competitive and consists of a few major players. Companies like Amgen Inc., Bluebird Bio, Gilead Sciences, Inc., Novartis AG, Orchard Therapeutics, Sibiono GeneTech Co. Ltd, Spark Therapeutics (Roche AG), and UniQure NV hold a substantial market share. They have various strategic alliances, such as collaborations and acquisitions, along with the launch of advanced products to secure their position in the global market.

Recent Developments

  • In June 2022, the European Medicine Agency granted conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) for the treatment of severe hemophilia A in adults. Roctavian is the first gene therapy to treat hemophilia A.
  • In February 2022, BioMarin Pharmaceutical Inc. reported updates on its investigational gene therapy programs in clinical development. The Company received additional requests from the Food and Drug Administration (FDA) for information needed to resolve the clinical hold of the PHEARLESS Phase 1/2 study of BMN 307 issued in September 2021.

Table of Contents


    1. 1.1 Study Assumptions and Market Definition

    2. 1.2 Scope of the Study




    1. 4.1 Market Overview

    2. 4.2 Market Drivers

      1. 4.2.1 Rising Investments in Research and Development

      2. 4.2.2 Technological Advancements

      3. 4.2.3 Growing Prevalence of Target Diseases like Cancer

    3. 4.3 Market Restraints

      1. 4.3.1 Lack of Standard Regulations

      2. 4.3.2 High Price of Products

    4. 4.4 Porter's Five Forces Analysis

      1. 4.4.1 Threat of New Entrants

      2. 4.4.2 Bargaining Power of Buyers/Consumers

      3. 4.4.3 Bargaining Power of Suppliers

      4. 4.4.4 Threat of Substitute Products

      5. 4.4.5 Intensity of Competitive Rivalry

  5. 5. MARKET SEGMENTATION (Market Size by Value- USD million)

    1. 5.1 By Indication

      1. 5.1.1 Cancer

      2. 5.1.2 Metabolic Disorders

      3. 5.1.3 Eye Disorders

      4. 5.1.4 Spinal Muscular Atrophy

      5. 5.1.5 Other Indications

    2. 5.2 By Technology

      1. 5.2.1 Adeno Virus Vector

      2. 5.2.2 Adeno-associated Virus Vector

      3. 5.2.3 Lentiviral Vector

      4. 5.2.4 Retroviral Vector

      5. 5.2.5 Herpes Virus Vector

      6. 5.2.6 Other Technologies

    3. 5.3 Geography

      1. 5.3.1 North America

        1. United States

        2. Canada

        3. Mexico

      2. 5.3.2 Europe

        1. Germany

        2. United Kingdom

        3. France

        4. Italy

        5. Spain

        6. Rest of Europe

      3. 5.3.3 Asia-Pacific

        1. China

        2. Japan

        3. India

        4. Australia

        5. South Korea

        6. Rest of Asia-Pacific

      4. 5.3.4 Middle-East and Africa

        1. GCC

        2. South Africa

        3. Rest of Middle-East and Africa

      5. 5.3.5 South America

        1. Brazil

        2. Argentina

        3. Rest of South America


    1. 6.1 Company Profiles

      1. 6.1.1 Amgen Inc.

      2. 6.1.2 Biogen Inc.

      3. 6.1.3 Bluebird Bio Inc.

      4. 6.1.4 Gilead Sciences Inc. (Kite Pharma)

      5. 6.1.5 Novartis AG

      6. 6.1.6 Orchard Therapeutics

      7. 6.1.7 Sibiono GeneTech Co. Ltd

      8. 6.1.8 F. Hoffmann-La Roche Ltd (Spark Therapeutics)

      9. 6.1.9 UniQure NV

      10. 6.1.10 Abeona Therapeutics Inc.

      11. 6.1.11 Generation Bio

      12. 6.1.12 Poseida Therapeutics

      13. 6.1.13 Astellas Pharma

      14. 6.1.14 Voyager Therapeutics Inc.

      15. 6.1.15 REGENXBIO Inc.

    2. *List Not Exhaustive

**Competitive Landscape covers- Business Overview, Financials, Products and Strategies and Recent Developments

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Frequently Asked Questions

The Global Gene Therapy Market market is studied from 2019 - 2027.

The Global Gene Therapy Market is growing at a CAGR of 25.71% over the next 5 years.

Asia Pacific is growing at the highest CAGR over 2021- 2026.

North America holds highest share in 2021.

Gilead Sciences, Inc., Novartis, Amgen Inc., Bluebird Bio, Biogen Inc are the major companies operating in Global Gene Therapy Market.

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