Global Gene Therapy for Mucopolysaccharidosis Market Research Report 2023

Mucopolysaccharidosis consists of a group of more than 40 genetic disorders that forms a part of lysosomal storage disease. They are caused by an inherited deficiency of an enzyme involved in the degradation of acid glycosaminoglycans (GAGs), which was previously known as mucopolysaccharidosis. All of the mucopolysaccharidoses are rare, autosomal recessive, except for mucopolysaccharidosis III, as it is X-linked.

The global Gene Therapy for Mucopolysaccharidosis market was valued at US$ million in 2022 and is anticipated to reach US$ million by 2029, witnessing a CAGR of % during the forecast period 2023-2029. The influence of COVID-19 and the Russia-Ukraine War were considered while estimating market sizes.

Over the past few years, gene therapy has emerged as one of the most exciting approaches for the treatment of mucopolysaccharidosis. One of the promising therapy is replacement gene therapy, which replaces faulty genes with normal ones in the body. It is considered to be a potential cure to mucopolysaccharidoses and a promising option in the long run.

This report aims to provide a comprehensive presentation of the global market for Gene Therapy for Mucopolysaccharidosis, with both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Gene Therapy for Mucopolysaccharidosis.

The Gene Therapy for Mucopolysaccharidosis market size, estimations, and forecasts are provided in terms of and revenue ($ millions), considering 2022 as the base year, with history and forecast data for the period from 2018 to 2029. This report segments the global Gene Therapy for Mucopolysaccharidosis market comprehensively. Regional market sizes, concerning products by type, by application, and by players, are also provided.

For a more in-depth understanding of the market, the report provides profiles of the competitive landscape, key competitors, and their respective market ranks. The report also discusses technological trends and new product developments.

The report will help the Gene Therapy for Mucopolysaccharidosis companies, new entrants, and industry chain related companies in this market with information on the revenues for the overall market and the sub-segments across the different segments, by company, by type, by application, and by regions.

1.1 Study Scope

1.2 Market Analysis by Type

1.2.1 Global Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Type: 2018 VS 2022 VS 2029

1.2.2 Intravenous

1.2.3 ICV

1.2.4 Intracerebral

1.2.5 Intracisternal

1.3 Market by Application

1.3.1 Global Gene Therapy for Mucopolysaccharidosis Market Growth by Application: 2018 VS 2022 VS 2029

1.3.2 Mucopolysaccharidosis I

1.3.3 Mucopolysaccharidosis II

1.3.4 Mucopolysaccharidosis III A

1.3.5 Mucopolysaccharidosis III B

1.4 Study Objectives

1.5 Years Considered

1.6 Years Considered

2.1 Global Gene Therapy for Mucopolysaccharidosis Market Perspective (2018-2029)

2.2 Gene Therapy for Mucopolysaccharidosis Growth Trends by Region

2.2.1 Global Gene Therapy for Mucopolysaccharidosis Market Size by Region: 2018 VS 2022 VS 2029

2.2.2 Gene Therapy for Mucopolysaccharidosis Historic Market Size by Region (2018-2023)

2.2.3 Gene Therapy for Mucopolysaccharidosis Forecasted Market Size by Region (2024-2029)

2.3 Gene Therapy for Mucopolysaccharidosis Market Dynamics

2.3.1 Gene Therapy for Mucopolysaccharidosis Industry Trends

2.3.2 Gene Therapy for Mucopolysaccharidosis Market Drivers

2.3.3 Gene Therapy for Mucopolysaccharidosis Market Challenges

2.3.4 Gene Therapy for Mucopolysaccharidosis Market Restraints

3.1 Global Top Gene Therapy for Mucopolysaccharidosis Players by Revenue

3.1.1 Global Top Gene Therapy for Mucopolysaccharidosis Players by Revenue (2018-2023)

3.1.2 Global Gene Therapy for Mucopolysaccharidosis Revenue Market Share by Players (2018-2023)

3.2 Global Gene Therapy for Mucopolysaccharidosis Market Share by Company Type (Tier 1, Tier 2, and Tier 3)

3.3 Players Covered: Ranking by Gene Therapy for Mucopolysaccharidosis Revenue

3.4 Global Gene Therapy for Mucopolysaccharidosis Market Concentration Ratio

3.4.1 Global Gene Therapy for Mucopolysaccharidosis Market Concentration Ratio (CR5 and HHI)

3.4.2 Global Top 10 and Top 5 Companies by Gene Therapy for Mucopolysaccharidosis Revenue in 2022

3.5 Gene Therapy for Mucopolysaccharidosis Key Players Head office and Area Served

3.6 Key Players Gene Therapy for Mucopolysaccharidosis Product Solution and Service

3.7 Date of Enter into Gene Therapy for Mucopolysaccharidosis Market

3.8 Mergers & Acquisitions, Expansion Plans

4.1 Global Gene Therapy for Mucopolysaccharidosis Historic Market Size by Type (2018-2023)

4.2 Global Gene Therapy for Mucopolysaccharidosis Forecasted Market Size by Type (2024-2029)

5.1 Global Gene Therapy for Mucopolysaccharidosis Historic Market Size by Application (2018-2023)

5.2 Global Gene Therapy for Mucopolysaccharidosis Forecasted Market Size by Application (2024-2029)

6.1 North America Gene Therapy for Mucopolysaccharidosis Market Size (2018-2029)

6.2 North America Gene Therapy for Mucopolysaccharidosis Market Growth Rate by Country: 2018 VS 2022 VS 2029

6.3 North America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023)

6.4 North America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029)

6.5 United States

6.6 Canada

7.1 Europe Gene Therapy for Mucopolysaccharidosis Market Size (2018-2029)

7.2 Europe Gene Therapy for Mucopolysaccharidosis Market Growth Rate by Country: 2018 VS 2022 VS 2029

7.3 Europe Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023)

7.4 Europe Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029)

7.5 Germany

7.6 France

7.7 U.K.

7.8 Italy

7.9 Russia

7.10 Nordic Countries

8.1 Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size (2018-2029)

8.2 Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Growth Rate by Region: 2018 VS 2022 VS 2029

8.3 Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size by Region (2018-2023)

8.4 Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size by Region (2024-2029)

8.5 China

8.6 Japan

8.7 South Korea

8.8 Southeast Asia

8.9 India

8.10 Australia

9.1 Latin America Gene Therapy for Mucopolysaccharidosis Market Size (2018-2029)

9.2 Latin America Gene Therapy for Mucopolysaccharidosis Market Growth Rate by Country: 2018 VS 2022 VS 2029

9.3 Latin America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023)

9.4 Latin America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029)

9.5 Mexico

9.6 Brazil

10.1 Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size (2018-2029)

10.2 Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Growth Rate by Country: 2018 VS 2022 VS 2029

10.3 Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023)

10.4 Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029)

10.5 Turkey

10.6 Saudi Arabia

10.7 UAE

11.1 Sangamo Therapeutics

11.1.1 Sangamo Therapeutics Company Detail

11.1.2 Sangamo Therapeutics Business Overview

11.1.3 Sangamo Therapeutics Gene Therapy for Mucopolysaccharidosis Introduction

11.1.4 Sangamo Therapeutics Revenue in Gene Therapy for Mucopolysaccharidosis Business (2018-2023)

11.1.5 Sangamo Therapeutics Recent Development

11.2 Swedish Orphan Biovitrum

11.2.1 Swedish Orphan Biovitrum Company Detail

11.2.2 Swedish Orphan Biovitrum Business Overview

11.2.3 Swedish Orphan Biovitrum Gene Therapy for Mucopolysaccharidosis Introduction

11.2.4 Swedish Orphan Biovitrum Revenue in Gene Therapy for Mucopolysaccharidosis Business (2018-2023)

11.2.5 Swedish Orphan Biovitrum Recent Development

11.3 uniQure

11.3.1 uniQure Company Detail

11.3.2 uniQure Business Overview

11.3.3 uniQure Gene Therapy for Mucopolysaccharidosis Introduction

11.3.4 uniQure Revenue in Gene Therapy for Mucopolysaccharidosis Business (2018-2023)

11.3.5 uniQure Recent Development

13.1 Research Methodology

13.1.1 Methodology/Research Approach

13.1.2 Data Source

13.2 Disclaimer

13.3 Author Details

List of Tables

    Table 1. Global Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Type (US$ Million): 2018 VS 2022 VS 2029

    Table 2. Key Players of Intravenous

    Table 3. Key Players of ICV

    Table 4. Key Players of Intracerebral

    Table 5. Key Players of Intracisternal

    Table 6. Global Gene Therapy for Mucopolysaccharidosis Market Size Growth by Application (US$ Million): 2018 VS 2022 VS 2029

    Table 7. Global Gene Therapy for Mucopolysaccharidosis Market Size by Region (US$ Million): 2018 VS 2022 VS 2029

    Table 8. Global Gene Therapy for Mucopolysaccharidosis Market Size by Region (2018-2023) & (US$ Million)

    Table 9. Global Gene Therapy for Mucopolysaccharidosis Market Share by Region (2018-2023)

    Table 10. Global Gene Therapy for Mucopolysaccharidosis Forecasted Market Size by Region (2024-2029) & (US$ Million)

    Table 11. Global Gene Therapy for Mucopolysaccharidosis Market Share by Region (2024-2029)

    Table 12. Gene Therapy for Mucopolysaccharidosis Market Trends

    Table 13. Gene Therapy for Mucopolysaccharidosis Market Drivers

    Table 14. Gene Therapy for Mucopolysaccharidosis Market Challenges

    Table 15. Gene Therapy for Mucopolysaccharidosis Market Restraints

    Table 16. Global Gene Therapy for Mucopolysaccharidosis Revenue by Players (2018-2023) & (US$ Million)

    Table 17. Global Gene Therapy for Mucopolysaccharidosis Market Share by Players (2018-2023)

    Table 18. Global Top Gene Therapy for Mucopolysaccharidosis Players by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in Gene Therapy for Mucopolysaccharidosis as of 2022)

    Table 19. Ranking of Global Top Gene Therapy for Mucopolysaccharidosis Companies by Revenue (US$ Million) in 2022

    Table 20. Global 5 Largest Players Market Share by Gene Therapy for Mucopolysaccharidosis Revenue (CR5 and HHI) & (2018-2023)

    Table 21. Key Players Headquarters and Area Served

    Table 22. Key Players Gene Therapy for Mucopolysaccharidosis Product Solution and Service

    Table 23. Date of Enter into Gene Therapy for Mucopolysaccharidosis Market

    Table 24. Mergers & Acquisitions, Expansion Plans

    Table 25. Global Gene Therapy for Mucopolysaccharidosis Market Size by Type (2018-2023) & (US$ Million)

    Table 26. Global Gene Therapy for Mucopolysaccharidosis Revenue Market Share by Type (2018-2023)

    Table 27. Global Gene Therapy for Mucopolysaccharidosis Forecasted Market Size by Type (2024-2029) & (US$ Million)

    Table 28. Global Gene Therapy for Mucopolysaccharidosis Revenue Market Share by Type (2024-2029)

    Table 29. Global Gene Therapy for Mucopolysaccharidosis Market Size by Application (2018-2023) & (US$ Million)

    Table 30. Global Gene Therapy for Mucopolysaccharidosis Revenue Market Share by Application (2018-2023)

    Table 31. Global Gene Therapy for Mucopolysaccharidosis Forecasted Market Size by Application (2024-2029) & (US$ Million)

    Table 32. Global Gene Therapy for Mucopolysaccharidosis Revenue Market Share by Application (2024-2029)

    Table 33. North America Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Country (US$ Million): 2018 VS 2022 VS 2029

    Table 34. North America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023) & (US$ Million)

    Table 35. North America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029) & (US$ Million)

    Table 36. Europe Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Country (US$ Million): 2018 VS 2022 VS 2029

    Table 37. Europe Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023) & (US$ Million)

    Table 38. Europe Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029) & (US$ Million)

    Table 39. Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Region (US$ Million): 2018 VS 2022 VS 2029

    Table 40. Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size by Region (2018-2023) & (US$ Million)

    Table 41. Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size by Region (2024-2029) & (US$ Million)

    Table 42. Latin America Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Country (US$ Million): 2018 VS 2022 VS 2029

    Table 43. Latin America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023) & (US$ Million)

    Table 44. Latin America Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029) & (US$ Million)

    Table 45. Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size Growth Rate by Country (US$ Million): 2018 VS 2022 VS 2029

    Table 46. Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size by Country (2018-2023) & (US$ Million)

    Table 47. Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size by Country (2024-2029) & (US$ Million)

    Table 48. Sangamo Therapeutics Company Detail

    Table 49. Sangamo Therapeutics Business Overview

    Table 50. Sangamo Therapeutics Gene Therapy for Mucopolysaccharidosis Product

    Table 51. Sangamo Therapeutics Revenue in Gene Therapy for Mucopolysaccharidosis Business (2018-2023) & (US$ Million)

    Table 52. Sangamo Therapeutics Recent Development

    Table 53. Swedish Orphan Biovitrum Company Detail

    Table 54. Swedish Orphan Biovitrum Business Overview

    Table 55. Swedish Orphan Biovitrum Gene Therapy for Mucopolysaccharidosis Product

    Table 56. Swedish Orphan Biovitrum Revenue in Gene Therapy for Mucopolysaccharidosis Business (2018-2023) & (US$ Million)

    Table 57. Swedish Orphan Biovitrum Recent Development

    Table 58. uniQure Company Detail

    Table 59. uniQure Business Overview

    Table 60. uniQure Gene Therapy for Mucopolysaccharidosis Product

    Table 61. uniQure Revenue in Gene Therapy for Mucopolysaccharidosis Business (2018-2023) & (US$ Million)

    Table 62. uniQure Recent Development

    Table 63. Research Programs/Design for This Report

    Table 64. Key Data Information from Secondary Sources

    Table 65. Key Data Information from Primary Sources

List of Figures

    Figure 1. Global Gene Therapy for Mucopolysaccharidosis Market Size Comparison by Type (2023-2029) & (US$ Million)

    Figure 2. Global Gene Therapy for Mucopolysaccharidosis Market Share by Type: 2022 VS 2029

    Figure 3. Intravenous Features

    Figure 4. ICV Features

    Figure 5. Intracerebral Features

    Figure 6. Intracisternal Features

    Figure 7. Global Gene Therapy for Mucopolysaccharidosis Market Size Comparison by Application (2023-2029) & (US$ Million)

    Figure 8. Global Gene Therapy for Mucopolysaccharidosis Market Share by Application: 2022 VS 2029

    Figure 9. Mucopolysaccharidosis I Case Studies

    Figure 10. Mucopolysaccharidosis II Case Studies

    Figure 11. Mucopolysaccharidosis III A Case Studies

    Figure 12. Mucopolysaccharidosis III B Case Studies

    Figure 13. Gene Therapy for Mucopolysaccharidosis Report Years Considered

    Figure 14. Global Gene Therapy for Mucopolysaccharidosis Market Size (US$ Million), Year-over-Year: 2018-2029

    Figure 15. Global Gene Therapy for Mucopolysaccharidosis Market Size, (US$ Million), 2018 VS 2022 VS 2029

    Figure 16. Global Gene Therapy for Mucopolysaccharidosis Market Share by Region: 2022 VS 2029

    Figure 17. Global Gene Therapy for Mucopolysaccharidosis Market Share by Players in 2022

    Figure 18. Global Top Gene Therapy for Mucopolysaccharidosis Players by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in Gene Therapy for Mucopolysaccharidosis as of 2022)

    Figure 19. The Top 10 and 5 Players Market Share by Gene Therapy for Mucopolysaccharidosis Revenue in 2022

    Figure 20. North America Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 21. North America Gene Therapy for Mucopolysaccharidosis Market Share by Country (2018-2029)

    Figure 22. United States Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 23. Canada Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 24. Europe Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 25. Europe Gene Therapy for Mucopolysaccharidosis Market Share by Country (2018-2029)

    Figure 26. Germany Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 27. France Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 28. U.K. Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 29. Italy Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 30. Russia Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 31. Nordic Countries Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 32. Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 33. Asia-Pacific Gene Therapy for Mucopolysaccharidosis Market Share by Region (2018-2029)

    Figure 34. China Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 35. Japan Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 36. South Korea Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 37. Southeast Asia Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 38. India Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 39. Australia Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 40. Latin America Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 41. Latin America Gene Therapy for Mucopolysaccharidosis Market Share by Country (2018-2029)

    Figure 42. Mexico Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 43. Brazil Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 44. Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 45. Middle East & Africa Gene Therapy for Mucopolysaccharidosis Market Share by Country (2018-2029)

    Figure 46. Turkey Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 47. Saudi Arabia Gene Therapy for Mucopolysaccharidosis Market Size YoY Growth (2018-2029) & (US$ Million)

    Figure 48. Sangamo Therapeutics Revenue Growth Rate in Gene Therapy for Mucopolysaccharidosis Business (2018-2023)

    Figure 49. Swedish Orphan Biovitrum Revenue Growth Rate in Gene Therapy for Mucopolysaccharidosis Business (2018-2023)

    Figure 50. uniQure Revenue Growth Rate in Gene Therapy for Mucopolysaccharidosis Business (2018-2023)

    Figure 51. Bottom-up and Top-down Approaches for This Report

    Figure 52. Data Triangulation

    Figure 53. Key Executives Interviewed