Global Gene Therapy for Rare Disease Market Growth, Share, Size, Trends and Forecast (2024 - 2030)

Segmented by ;

Approved Drugs - Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna) and Strimvelis, Pipeline Drugs - GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061 and Others.

Segmented by Therapeutic Application;

Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders and Others.

Segmented by Geography;

North America, Europe, Asia Pacific, Middle East and Africa and Latin America - Report Timeline (2020 - 2030).
Report ID: Rn149287675 Published Date: April, 2024 Updated Date: May, 2024

Introduction

Global Gene Therapy for Rare Disease Market (USD Million), 2020 - 2030

*Graph shown for illustrative purpose only, the final report will have the graph with actual data and data labels
# Global Market (USD Million), 2020 - 2030
2020 108.6
2021 121.6
2022 145.7
2023 153.5
2024 169.9
2025 170.7
2026 191.2
2027 218.3
2028 221.5
2029 246.6
2030 265.6

In the year 2023, the Global Gene Therapy for Rare Disease Market was valued at USD 1,991.48 million. The size of this market is expected to increase to USD 9,602.96 million by the year 2030, while growing at a Compounded Annual Growth Rate (CAGR) of 25.2%.

  1. Introduction
    1. Research Objectives and Assumptions
    2. Research Methodology
    3. Abbreviations
  2. Market Definition & Study Scope
  3. Executive Summary
    1. Market Snapshot, By By Drug
    2. Market Snapshot, By Therapeutic Application
    3. Market Snapshot, By Region
    4. Market Opportunity Map
  4. Global Gene Therapy for Rare Disease Market Dynamics
    1. Drivers, Restraints and Opportunities
      1. Drivers
      2. Restraints
      3. Opportunities
    2. Regulatory Scenario
    3. Reimbursement Scenario
    4. Mergers & Acquisitions, Collaborations & Partnerships, Agreements
    5. Pipeline Analysis/Technological Advancements
    6. PEST Analysis
      1. Political Analysis
      2. Economic Analysis
      3. Social Analysis
      4. Technological Analysis
    7. Porter's Analysis
      1. Bargaining Power of Suppliers
      2. Bragaining Power of Buyers
      3. Threat of Substitutes
      4. Threat of New Entrants
      5. Industry Rivalry
  5. Market Segmentation
    1. Global Gene Therapy for Rare Disease Market, By By Drug, 2022- 2032 (USD Million)
      1. Introduction
      2. Market Share Analysis, 2024 and 2030 (%)
      3. Y-o-Y Growth Analysis, 2021 - 2030
      4. Segment Trends
        1. Approved Drugs
          1. Introduction
          2. Market Share Analysis, 2024 and 2030 (%)
          3. Y-o-Y Growth Analysis, 2021 - 2030
          4. Segment Trends
            1. Tisagenlecleucel (Kymriah)
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            2. Axicabtagene ciloleucel (Yescarta)
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            3. Voretigene neparvovec (Luxturna)
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            4. Strimvelis
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        2. Pipeline Drugs
          1. Introduction
          2. Market Share Analysis, 2024 and 2030 (%)
          3. Y-o-Y Growth Analysis, 2021 - 2030
          4. Segment Trends
            1. GT-AADC
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            2. Fidanacogene elaparvovec (SPK-9011)
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            3. OTL-200
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            4. bb2121
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            5. AMT-061
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
            6. Others
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
    2. Global Gene Therapy for Rare Disease Market, By Therapeutic Application, 2022- 2032 (USD Million)
      1. Introduction
      2. Market Share Analysis, 2024 and 2030 (%)
      3. Y-o-Y Growth Analysis, 2021 - 2030
      4. Segment Trends
        1. Oncology
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        2. Neurological Disorders
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        3. Ophthalmic Disorders
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        4. Hematological Disorders
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        5. Immunodeficiency Disorders
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        6. Metabolic Disorders
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
        7. Others
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, 2020 - 2030 (USD Million)
    3. Global Gene Therapy for Rare Disease Market, By Geography, 2022- 2032 (USD Million)
      1. Introduction
      2. Market Share Analysis, 2024 and 2030 (%)
      3. Y-o-Y Growth Analysis, 2021 - 2030
      4. Regional Trends
        1. North America
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
          3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            1. United States
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            2. Canada
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
        2. Europe
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
          3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            1. Germany
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            2. United Kingdom
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            3. France
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            4. Italy
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            5. Spain
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            6. Rest of Europe
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
        3. Asia Pacific
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
          3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            1. Japan
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            2. China
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            3. India
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            4. Australia & New Zealand
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            5. South Korea
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            6. ASEAN (Association of South East Asian Countries)
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            7. Rest of Asia Pacific
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
        4. Middle East & Africa
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
          3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            1. GCC
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            2. Israel
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            3. South Africa
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            4. Rest of Middle East & Africa
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
        5. Latin America
          1. Introduction
          2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
          3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            1. Brazil
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            2. Mexico
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            3. Argentina
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
            4. Rest of Latin America
              1. Introduction
              2. Market Size and Forecast, and Y-o-Y Growth, By , 2020 - 2030 (USD Million)
              3. Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2020 - 2030 (USD Million)
  6. Competitive Landscape
    1. Heat Map Analysis
    2. Company Profiles
      1. BioMarin Pharmaceutical Inc.
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      2. PTC Therapeutics Inc.
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      3. Orchard Therapeutics Plc.
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      4. uniQure N.V
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      5. Spark Therapeutics, Inc.
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      6. Bluebird Bio, Inc.
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      7. Juno Therapeutics Inc. (Celgene Corporation)
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
      8. Novartis International AG
        1. Company Overview and Product Portfolio
        2. Key Developments
        3. Financial Overview
        4. Strategies
        5. Company SWOT Analysis
  7. Analyst Views
  8. Future Outlook of the Market

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