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Market Research Report

GENE THERAPY FOR RARE DISEASE MARKET SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2026 - 2033)

Segmentation
  • By DrugApproved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and Strimvelis) and Pipeline Drugs (GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and Others)
  • By Therapeutic ApplicationOncology · Neurological Disorders · Ophthalmic Disorders · Hematological Disorders · Immunodeficiency Disorders · Metabolic Disorders · and Others
  • By GeographyNorth America · Europe · Asia Pacific · Latin America · Middle East · and Africa
  • Published In01 Jun 2026
  • Report CodeCMI2321
  • Pages250+
  • FormatsExcel and PDF
  • Base Year2025
  • Estimated Year2026
  • Historical Range2020 - 2024
  • Forecast Period2026 - 2033
  1. Research Objectives and Assumptions

    • Research Objectives
    • Assumptions
    • Abbreviations
  2. Market Purview

    • Report Description
      • Market Definition and Scope
    • Executive Summary
      • Market Snapshot, By Drug
      • Market Snapshot, By Therapeutic Application
      • Market Snapshot, By Region
    • Coherent Opportunity Map (COM)
  3. Market Dynamics, Regulations, and Trends Analysis

    • Market Dynamics
      • Drivers
      • Restraints
      • Market Opportunities
    • Impact Analysis
    • Market Trends
    • Key Developments
    • Product Approval
    • Collaboration/Merge/Acquisition
    • Regulatory Landscape
    • PEST Analysis
    • Porter’s Five Forces Analysis
    • Epidemiology
    • Robust Pipeline Analysis
  4. Global Gene Therapy for Rare Disease Market, By Drug, 2021 – 2033, (USD Mn)

    • Introduction
      • Market Share Analysis, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, 2022– 2033
      • Segment Trends
    • Approved Drugs
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
        • Tisagenlecleucel (Kymriah)
        • Axicabtagene ciloleucel (Yescarta)
        • Voretigene neparvovec (Luxturna)
        • Strimvelis
    • Pipeline Drugs
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
        • GT-AADC
        • Fidanacogene elaparvovec (SPK-9011)
        • OTL-200
        • bb2121
        • AMT-061
        • Others
  5. Global Gene Therapy for Rare Disease Market, By Therapeutic Application, 2021 – 2033, (USD Mn)

    • Introduction
      • Market Share Analysis, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, 2022 – 2033
      • Segment Trends
    • Oncology
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Neurological Disorders
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Ophthalmic Disorders
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Hematological Disorders
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Immunodeficiency Disorders
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Metabolic Disorders
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Others
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
  6. Global Gene Therapy for Rare Disease Market, By Region, 2021 – 2033, (USD Mn)

    • Introduction
      • Market Share Analysis, By Region, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, For Regions, 2022–2033
      • Regional Trends
    • North America
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • U.S.
        • Canada
    • Latin America
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • U.K.
        • Germany
        • Italy
        • France
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • China
        • India
        • Japan
        • ASEAN
        • Australia
        • South Korea
        • Rest of Asia Pacific
    • Middle East
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Therapeutic Application, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • North Africa
        • Central Africa
        • South Africa
  7. Competitive Landscape

    • Heat Map Analysis
    • Market Share Analysis
    • Company Profile
      • Kite Pharma, Inc. (Gilead Sciences, Inc.)
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Novartis International AG
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Juno Therapeutics Inc. (Celgene Corporation)
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Bluebird Bio, Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Spark Therapeutics, Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • uniQure N.V.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Orchard Therapeutics Plc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • PTC Therapeutics, Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      •  BioMarin Pharmaceutical Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
    • Analyst Views
  8. Section

    • References
    • Research Methodology
    • About Us