+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

Gene Therapy Market by Indication (Inherited Retinal Disease, Acute Lymphoblastic Leukemia, ADA-SCID), by Vector Type, and by Region - Global Forecast to 2023-2033

  • PDF Icon

    Report

  • 200 Pages
  • January 2023
  • Region: Global
  • Quintana Research and Consulting
  • ID: 5717224
The gene therapy market size is estimated to be USD 7,652.4 million in 2022 and is expected to witness a CAGR of 19.41% during the forecast period 2023-2033. Rapid technological developments in cellular and molecular biology and surge in the number of cancer cases and other chronic diseases worldwide are projected to drive the market growth in near future. Furthermore, rise in funding and investments in this industry and strong regulatory assistance are other factors supporting the market growth. However, high price of product development and stringent management methods are predicted to limit market growth.

Rise in funding and investments in this industry is expected to fuel the market growth during the projected period. Additionally, it is anticipated that Rise in funding and investments in this industry will offer market participants significant growth potential. For the launch of innovative products, several biopharma companies are making investments in the field. For instance, Ori Biotech raised more than USD 100.0 million in Series B funding in January 2022 to launch advanced platform for researching cell and gene therapies. From pre-commercialization through market launch, this funding enabled a quick transition.

Strong regulatory assistance is expected to propel the market growth during the forecast period. The methods used by a number of global regulatory agencies to advertise treatments have improved. One of the instances is the FDA's assistance for CAR-T technology. Regulators are permitting flexibility in the conventional hierarchy of how clinical trials are conducted, particularly in phase II and III research. In addition, the FDA anticipated that by 2025, 10 to 20 novel cell and gene therapies would be approved annually.

Segmentation

By Indication

The market is categorized into inherited retinal disease, acute lymphoblastic leukemia (ALL), ADA-SCID, large b-cell lymphoma, melanoma (lesions), head & neck squamous cell carcinoma, beta-thalassemia major/SCD, spinal muscular atrophy (SMA), peripheral arterial disease, others. In 2022, the spinal muscular atrophy (SMA) segment accounted for the highest revenue share due to the fact autosamplers being used more frequently due to the fact that SMA is one of the most frequent fatal hereditary disorders of infancy even though it is a rare disorder. The development of Zolgensma (AVXS-101) has shown its capability to effectively treat SMA and modify the phenotype of the disease. T the FDA granted approval for Zolgensma, a drug developed by Novartis that targets the root cause of SMA. The only gene therapy in this area to date receive approval is Zolgensma. The approval of this gene therapy is indication that treatments for severe genetic diseases including SMA are becoming common. Inherited retinal disease is anticipated to experience a high CAGR during the projection period. The market has been fueled by an increase in R&D efforts for the development of gene therapies used to treat ocular disorders, particularly hereditary retinal disease.

By Vector Type

The market is categorized into AAV, lentivirus, modified herpes simplex virus, retrovirus & gamma retrovirus, non-viral plasmid vector, adenovirus, others. In the global market, the retrovirus & gamma retrovirus segment accounted for the largest revenue share in 2022 owing to simplicity of DNA incorporation and isolation. The two copies of single-stranded RNA that are produced by retroviruses can be changed into double-stranded DNA, which can then be incorporated into the chromosomes of the host cell. The use of retroviruses is a primary focus of many ongoing clinical investigations. Adeno-Associated Viral (AAV) vectors are anticipated to demonstrate a large market share in the upcoming years. AAVs are in high demand, and their usage in clinical studies is expanding annually due to their high efficacy in delivering the gene to the region of interest. The growth is a result of clinical trials related to the advancement of gene therapy treatments for orthopedic and ophthalmic diseases that have shown enhanced efficiency and efficacy. Recent years have witnessed a significant rise in the use of AAV across many therapeutic sectors, adoption rates are expected to considerably increase over the next few years.

Regional Markets

In 2022, North America region accounted for the highest revenue in the gene therapy market and is expected to maintain its dominance during the forecast period. This is attributed to rising R&D expenditures from both large and small companies on the development of drugs for optimum therapy. The market is also being driven by the governments' growing investment levels and the rising incidence of certain diseases. The Spinal Muscular Atrophy Foundation estimates that in 2020, spinal muscular atrophy would affect between 10,000 and 25,000 children and adults in the United States, making it one of the prevalent rare diseases. Asia Pacific is anticipated to have the fastest rate of growth during the projected period. This is due to the country's sizable population's unmet medical demands and rising demand for novel technology in the management of uncommon but steadily escalating diseases. The Asia Pacific market for the commercial use of genetic therapies is predicted to have considerable expansion over the projected period due to the easy access to resources, the presence of big corporations locally, and rising government funding.

Competitor Insights

Some of the key players operating in the gene therapy market are Oxford BioMedica plc, Dimension Therapeutics, Inc., Novartis AG, Sangamo Therapeutics, Inc., REGENXBIO, Inc., Sibiono GeneTech Co., Ltd., UniQure N.V., Transgene, Calimmune, Inc., Bristol-Myers Squibb Company, Applied Genetic Technologies Corp, F. Hoffmann-La Roche Ltd., Bluebird Bio, Inc., Shire Plc, American Gene Technologies, Cellectis S.A., Orchard Therapeutics, Gilead Lifesciences, Inc., Benitec Biopharma Ltd., Epeius Biotechnologies Corp., Gensight Biologics S.A., SANOFI, Shanghai Sunway Biotech Co., Ltd., Astellas Pharma, Inc., Taxus Cardium Pharmaceuticals Group, Inc. (Gene Biotherapeutics), BioMarin Pharmaceuticals, Inc.

To achieve a competitive edge, these companies are implementing a variety of growth strategies. Strategic partnerships, alliances, and agreements with different businesses and research institutions are seen to be the main strategies used by companies in this sector.

For instance, the American biotech startup 64x Bio raised USD 55.0 million in funding in January 2022 to improve its manufacturing platform for gene therapy.

The company's VectorSelect platform was projected to grow as a result of this initiative. Additionally, Pfizer, Inc. stated in April 2022 that it would open its first location in the United States for the Phase 3 trials of investigational gene therapy for people with Duchenne muscular dystrophy. The global Phase 3 CIFFREO study was begun after receiving regulatory agency approval from Canada, Taiwan, the UK, Spain, and Belgium.This comprehensive research report focuses on the global and regional market size and forecasts of diverse segments including indication and vector from 2022 to 2033.

Segmentation: Gene Therapy Market Report 2022 - 2033

Indication (Revenue, USD Million), 2022 - 2033

  • Inherited Retinal Disease
  • Acute Lymphoblastic Leukemia (ALL)
  • ADA-SCID
  • Large B-cell Lymphoma
  • Melanoma (lesions)
  • Head & Neck Squamous Cell Carcinoma
  • Beta-Thalassemia Major/SCD
  • Spinal Muscular Atrophy (SMA)
  • Peripheral arterial disease
  • Others

Vector (Revenue, USD Million), 2022 - 2033

  • AAV
  • Lentivirus
  • Modified Herpes Simplex Virus
  • Retrovirus & gamma Retrovirus
  • Non-viral Plasmid Vector
  • Adenovirus
  • Others

By Region (Revenue, USD Million), 2022 - 2033

North America

  • U.S.
  • Canada

Europe

  • Germany
  • France
  • UK
  • Spain
  • Italy
  • Rest of Europe

Asia Pacific

  • China
  • Japan
  • India
  • Australia
  • South Korea
  • Rest of APAC

Latin America

  • Brazil
  • Mexico
  • Argentina
  • Rest of LATAM

Middle East & Africa

  • South Africa
  • GCC
  • Rest of MEA

Table of Contents

1. Research Methodology
1.1. Study Objectives
1.2. Study Scope
1.3. Research Framework
1.3.1. Research Models
1.3.1.1. Bottom-up Approach
1.3.1.2. Top-down Approach
1.3.1.3. Data Triangulation
1.3.2. Data Analysis
1.3.2.1. Data Validation
1.3.2.2. Market Size Estimation
1.3.2.3. Market Forecast Model

2. Introduction: Gene Therapy

3. Executive Summary

4. Market Dynamics
4.1. Market Drivers
4.1.1. Driver 1
4.1.2. Driver 2
4.1.3. Driver 3
4.1.4. Driver 4
4.1.5. Driver 5
4.2. Market Restraint
4.2.1. Restraint 1
4.2.2. Restraint 2
4.2.3. Restraint 3
4.2.4. Restraint 4
4.3. Market Opportunities
4.3.1. Opportunity 1
4.3.2. Opportunity 2
4.3.3. Opportunity 3
4.3.4. Opportunity 4
4.4. Market Trends
4.4.1. Trend 1
4.4.2. Trend 2
4.4.3. Trend 3
4.4.4. Trend 4
4.5. Market Challenges
4.5.1. Challenge 1
4.5.2. Challenge 2
4.5.3. Challenge 3
4.5.4. Challenge 4

5. Market Environment Analysis
5.1. Porter’s 5 Forces Analysis
5.2. PESTEL Analysis
5.3. SWOT Analysis

6. COVID-19 Impact Analysis: Gene Therapy Market

7. Market Analysis by Indication
7.1. Inherited Retinal Disease
7.1.1. Inherited Retinal Disease Market Forecast, 2022-2033 (USD Million)
7.2. Acute Lymphoblastic Leukemia (ALL)
7.2.1. Acute Lymphoblastic Leukemia (ALL) Market Forecast, 2022-2033 (USD Million)
7.3. ADA-SCID
7.3.1. ADA-SCID Market Forecast, 2022-2033 (USD Million)
7.4. Large B-cell Lymphoma
7.4.1. Large B-cell Lymphoma Market Forecast, 2022-2033 (USD Million)
7.5. Melanoma (lesions)
7.5.1. Melanoma (lesions) Market Forecast, 2022-2033 (USD Million)
7.6. Head & Neck Squamous Cell Carcinoma
7.6.1. Head & Neck Squamous Cell Carcinoma Market Forecast, 2022-2033 (USD Million)
7.7. Beta-Thalassemia Major/SCD
7.7.1. Beta-Thalassemia Major/SCD Market Forecast, 2022-2033 (USD Million)
7.8. Spinal Muscular Atrophy (SMA)
7.8.1. Spinal Muscular Atrophy (SMA) Market Forecast, 2022-2033 (USD Million)
7.9. Peripheral arterial disease
7.9.1. Peripheral arterial disease Market Forecast, 2022-2033 (USD Million)
7.10. Others
7.10.1. Others Market Forecast, 2022-2033 (USD Million)

8. Market Analysis by Vector
8.1. AAV
8.1.1. AAV Market Forecast, 2022-2033 (USD Million)
8.2. Lentivirus
8.2.1. Lentivirus Market Forecast, 2022-2033 (USD Million)
8.3. Modified Herpes Simplex Virus
8.3.1. Modified Herpes Simplex Virus Market Forecast, 2022-2033 (USD Million)
8.4. Retrovirus & gamma Retrovirus
8.4.1. Retrovirus & gamma Retrovirus Market Forecast, 2022-2033 (USD Million)
8.5. Non-viral Plasmid Vector
8.5.1. Non-viral Plasmid Vector Market Forecast, 2022-2033 (USD Million)
8.6. Adenovirus
8.6.1. Adenovirus Market Forecast, 2022-2033 (USD Million)
8.7. Others
8.7.1. Others Market Forecast, 2022-2033 (USD Million)

9. Regional Market Analysis
9.1. Regional Market Trends

10. North America Gene Therapy Market
10.1. North America Gene Therapy Market
10.1.1. North America Market Size and Forecast, 2022-2033 (USD Million)
10.1.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
10.1.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
10.2. U.S. Gene Therapy Market
10.2.1. U.S. Market Size and Forecast, 2022-2033 (USD Million)
10.2.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
10.2.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
10.3. Canada Gene Therapy Market
10.3.1. Canada Market Size and Forecast, 2022-2033 (USD Million)
10.3.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
10.3.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)

11. Europe Gene Therapy Market
11.1. Europe Gene Therapy Market
11.1.1. Europe Market Size and Forecast, 2022-2033 (USD Million)
11.1.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.1.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
11.2. Germany Gene Therapy Market
11.2.1. Germany Market Size and Forecast, 2022-2033 (USD Million)
11.2.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.2.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
11.3. UK Gene Therapy Market
11.3.1. UK Market Size and Forecast, 2022-2033 (USD Million)
11.3.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.3.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
11.4. France Gene Therapy Market
11.4.1. France Market Size and Forecast, 2022-2033 (USD Million)
11.4.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.4.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
11.5. Spain Gene Therapy Market
11.5.1. Spain Market Size and Forecast, 2022-2033 (USD Million)
11.5.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.5.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
11.6. Italy Gene Therapy Market
11.6.1. Italy Market Size and Forecast, 2022-2033 (USD Million)
11.6.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.6.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
11.7. Rest of Europe Gene Therapy Market
11.7.1. Rest of Europe Market Size and Forecast, 2022-2033 (USD Million)
11.7.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
11.7.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)

12. Asia Pacific Gene Therapy Market
12.1. Asia Pacific Gene Therapy Market
12.1.1. Asia Pacific Market Size and Forecast, 2022-2033 (USD Million)
12.1.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.1.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
12.2. Japan Gene Therapy Market
12.2.1. Japan Market Size and Forecast, 2022-2033 (USD Million)
12.2.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.2.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
12.3. China Gene Therapy Market
12.3.1. China Market Size and Forecast, 2022-2033 (USD Million)
12.3.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.3.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
12.4. India Gene Therapy Market
12.4.1. India Market Size and Forecast, 2022-2033 (USD Million)
12.4.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.4.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
12.5. South Korea Gene Therapy Market
12.5.1. South Korea Market Size and Forecast, 2022-2033 (USD Million)
12.5.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.5.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
12.5.1.3. Market Size and Forecast by Device, 2022-2033 (USD Million)
12.6. Australia Gene Therapy Market
12.6.1. Australia Market Size and Forecast, 2022-2033 (USD Million)
12.6.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.6.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
12.7. Rest of Asia Pacific Gene Therapy Market
12.7.1. Rest of Asia Pacific Market Size and Forecast, 2022-2033 (USD Million)
12.7.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
12.7.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)

13. Latin America Gene Therapy Market
13.1. Latin America Gene Therapy Market
13.1.1. Latin America Market Size and Forecast, 2022-2033 (USD Million)
13.1.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
13.1.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
13.2. Brazil Gene Therapy Market
13.2.1. Brazil Market Size and Forecast, 2022-2033 (USD Million)
13.2.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
13.2.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
13.3. Mexico Gene Therapy Market
13.3.1. Mexico Market Size and Forecast, 2022-2033 (USD Million)
13.3.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
13.3.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
13.4. Argentina Gene Therapy Market
13.4.1. Argentina Market Size and Forecast, 2022-2033 (USD Million)
13.4.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
13.4.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
13.5. Rest of Latin America Gene Therapy Market
13.5.1. Rest of Latin America Market Size and Forecast, 2022-2033 (USD Million)
13.5.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
13.5.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)

14. MEA Gene Therapy Market
14.1. MEA Gene Therapy Market
14.1.1. MEA Market Size and Forecast, 2022-2033 (USD Million)
14.1.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
14.1.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
14.2. GCC Gene Therapy Market
14.2.1. GCC Market Size and Forecast, 2022-2033 (USD Million)
14.2.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
14.2.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
14.3. South Africa Gene Therapy Market
14.3.1. South Africa Market Size and Forecast, 2022-2033 (USD Million)
14.3.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
14.3.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)
14.4. Rest of MEA Gene Therapy Market
14.4.1. Rest of MEA Market Size and Forecast, 2022-2033 (USD Million)
14.4.1.1. Market Size and Forecast by Indication, 2022-2033 (USD Million)
14.4.1.2. Market Size and Forecast by Vector, 2022-2033 (USD Million)

15. Competitor Analysis
15.1. Market Share Analysis, 2022
15.2. Major Recent Developments, 2019-2022

16. Company Profiles
16.1. Oxford BioMedica plc
16.1.1. Company Snapshot
16.1.2. Company Overview
16.1.3. Financials
16.1.4. Product Benchmarking
16.1.5. Recent Developments
16.2. Dimension Therapeutics, Inc.
16.2.1. Company Snapshot
16.2.2. Company Overview
16.2.3. Financials
16.2.4. Product Benchmarking
16.2.5. Recent Developments
16.3. Novartis AG
16.3.1. Company Snapshot
16.3.2. Company Overview
16.3.3. Financials
16.3.4. Product Benchmarking
16.3.5. Recent Developments
16.4. Sangamo Therapeutics, Inc.
16.4.1. Company Snapshot
16.4.2. Company Overview
16.4.3. Financials
16.4.4. Product Benchmarking
16.4.5. Recent Developments
16.5. Sibiono GeneTech Co., Ltd.
16.5.1. Company Snapshot
16.5.2. Company Overview
16.5.3. Financials
16.5.4. Product Benchmarking
16.5.5. Recent Developments
16.6. UniQure N. V.
16.6.1. Company Snapshot
16.6.2. Company Overview
16.6.3. Financials
16.6.4. Product Benchmarking
16.6.5. Recent Developments
16.7. UniQure N. V.
16.7.1. Company Snapshot
16.7.2. Company Overview
16.7.3. Financials
16.7.4. Product Benchmarking
16.7.5. Recent Developments
16.8. Transgene
16.8.1. Company Snapshot
16.8.2. Company Overview
16.8.3. Financials
16.8.4. Product Benchmarking
16.8.5. Recent Developments
16.9. Calimmune, Inc.
16.9.1. Company Snapshot
16.9.2. Company Overview
16.9.3. Financials
16.9.4. Product Benchmarking
16.9.5. Recent Developments
16.10. Bristol-Myers Squibb Company
16.10.1. Company Snapshot
16.10.2. Company Overview
16.10.3. Financials
16.10.4. Product Benchmarking
16.10.5. Recent Developments
16.11. Applied Genetic Technologies Corp
16.11.1. Company Snapshot
16.11.2. Company Overview
16.11.3. Financials
16.11.4. Product Benchmarking
16.11.5. Recent Developments
16.12. F. Hoffmann-La Roche Ltd.
16.12.1. Company Snapshot
16.12.2. Company Overview
16.12.3. Financials
16.12.4. Product Benchmarking
16.12.5. Recent Developments
16.13. Bluebird Bio, Inc.
16.13.1. Company Snapshot
16.13.2. Company Overview
16.13.3. Financials
16.13.4. Product Benchmarking
16.13.5. Recent Developments
16.14. Shire Plc
16.14.1. Company Snapshot
16.14.2. Company Overview
16.14.3. Financials
16.14.4. Product Benchmarking
16.14.5. Recent Developments
16.15. American Gene Technologies, Cellectis S. A.
16.15.1. Company Snapshot
16.15.2. Company Overview
16.15.3. Financials
16.15.4. Product Benchmarking
16.15.5. Recent Developments
16.16. Others Prominent Players

17. Conclusion & Recommendations

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Oxford BioMedica plc
  • Dimension Therapeutics Inc.
  • Novartis AG
  • Sangamo Therapeutics Inc.
  • REGENXBIO Inc.
  • Sibiono GeneTech Co. Ltd.
  • UniQure N.V.
  • Transgene
  • Calimmune Inc.
  • Bristol-Myers Squibb Company
  • Applied Genetic Technologies Corp
  • F. Hoffmann-La Roche Ltd.
  • Bluebird Bio Inc.
  • Shire Plc
  • American Gene Technologies
  • Cellectis S.A.
  • Orchard Therapeutics
  • Gilead Lifesciences Inc.
  • Benitec Biopharma Ltd.
  • Epeius Biotechnologies Corp.
  • Gensight Biologics S.A.
  • SANOFI
  • Shanghai Sunway Biotech Co. Ltd.
  • Astellas Pharma Inc.
  • Taxus Cardium Pharmaceuticals Group Inc. (Gene Biotherapeutics)
  • BioMarin Pharmaceuticals Inc.