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

Gene Therapy for Rare Disease Market, By Drug (Approved 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 Application (Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), By Geography (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)

Published In: 01 Jun, 2026
Code: CMI2321
Page number: 250+
Formats: Excel and PDF
Industry: Biotechnology
- Historical Range : 2020 - 2024
- Base Year : 2025
- Estimated Year : 2026
- Forecast Period : 2026 - 2033

Table of Contents (Version-2026)

Research Objectives and Assumptions
- Research Objectives
- Assumptions
- Abbreviations
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)
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
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
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)
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
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
Section
- References
- Research Methodology
- About Us

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