罕見疾病基因治療市場：依藥物、治療應用和地區分類。

Gene Therapy for Rare Disease Market, By Drug, By Therapeutic Application, By Geography

出版日期: 2026年06月01日 | 出版商:

Coherent Market Insights | 英文 250+ Pages | 商品交期: 2-3個工作天內

價格

簡介目錄

預計到2026年，罕見疾病基因治療市場規模將達到2.425億美元，2033年將達到19.787億美元。預計從2026年到2033年，該市場將以35%的複合年成長率成長。

分析範圍		分析詳情
基準年	2025	市場規模（2026 年）	2.425億美元
效能數據	2020-2024	預測期	2026-2033
預測期（2026-2033 年）複合年成長率	35.00%	預計金額（2033 年）	19.787億美元

近年來，許多基因療法被認定為孤兒藥。孤兒藥通常是指根據1983年《孤兒藥法案》獲準用於一種或多種適應症的藥物。該法案旨在支持為罕見疾病患者開發創新療法。 1983年《孤兒藥法案》的頒佈建立了孤兒藥認定體系，加速了罕見疾病療法的研發與批准。 2017年和2018年，獲準的孤兒藥數量和適應症數量均創歷史新高。基因療法的生產需要最尖端科技、高昂的研發成本以及經驗豐富的科學家和研究人員的參與，這也反映在這些療法的高昂價格上。

市場動態

市場上主要的參與者正致力於開發亨丁頓舞蹈症等新型適應症的基因療法，目前尚無針對亨丁頓舞蹈症的認證核可藥。例如，uniQure NV公司正在開發AMT-130，這是一種用於治療亨丁頓舞蹈症（HD）的新型基因療法。亨丁頓舞蹈症是一種罕見且致命的神經退化遺傳疾病。 AMT-130目前正處於臨床前試驗階段，預計不久將獲得批准。此外，一些公司正在開發針對血友病B和血友病A等罕見疾病的基因療法，這些療法有望實現長期治療，並有可能取代這些疾病的終身用藥。預計這些新型療法在不久的將來推向市場，將為市場成長創造巨大且極具前景的機會。除了擴大健保報銷範圍外，製造商還在推出「按療效付費」等創新支付方式。然而，這些療法的高昂成本以及在亞太地區和拉丁美洲等新興經濟體商業化方面可能面臨的挑戰，可能會抑制市場成長。

本報告的主要特點

本報告揭示了各個細分市場的潛在商機，並描述了該市場中一系列有吸引力的投資提案。
此外，它還提供了有關市場促進因素和限制因素、機會、新產品發布和批准、市場趨勢、區域展望以及主要公司採取的競爭策略的重要見解。
本節根據公司概況、產品系列、主要趨勢、財務表現和策略等各種參數，對全球罕見疾病基因治療市場的主要公司進行介紹。
本報告中的見解將使負責人和企業經營團隊能夠就未來的產品發布、技術更新、市場擴張和行銷策略做出明智的決策。
《全球罕見疾病基因治療市場》報告面向該行業的眾多相關人員，包括投資者、供應商、產品製造商、經銷商、新參與企業和金融分析師。
相關人員可以透過分析全球罕見疾病基因治療市場時所使用的各種策略矩陣來促進決策。

北美洲
- 美國
- 加拿大
拉丁美洲
- 巴西
- 墨西哥
- 阿根廷
- 其他拉丁美洲國家
歐洲
- 英國
- 德國
- 義大利
- 法國
- 西班牙
- 俄羅斯
- 其他歐洲國家
亞太地區
- 中國
- 印度
- 日本
- ASEAN
- 澳洲
- 韓國
- 其他亞太國家
中東
- GCC
- 以色列
- 其他中東國家
非洲
- 北非
- 中非
- 南部非洲

第7章競爭情勢

熱圖分析
市佔率分析
公司簡介
- Kite Pharma, Inc.（Gilead Sciences, Inc.）
- Novartis International AG
- Juno Therapeutics Inc.（Celgene Corporation）
- Bluebird Bio, Inc.
- Spark Therapeutics, Inc.
- uniQure NV
- Orchard Therapeutics Plc.
- PTC Therapeutics, Inc.
- BioMarin Pharmaceutical Inc.
分析師意見

第8章

參考
分析方法
關於 Coherent Market Insights

簡介目錄

Product Code: CMI2321

Gene Therapy for Rare Disease Market is estimated to be valued at USD 242.5 Mn in 2026 and is expected to reach USD 1,978.7 Mn by 2033, growing at a compound annual growth rate (CAGR) of 35% from 2026 to 2033.

Report Coverage		Report Details
Base Year:	2025	Market Size in 2026:	USD 242.5 Mn
Historical Data for:	2020 To 2024	Forecast Period:	2026 To 2033
Forecast Period 2026 to 2033 CAGR:	35.00%	2033 Value Projection:	USD 1,978.7 Mn

In recent past, most of the gene therapies received orphan drug designations. Orphan drugs are generally defined as those medicines with one or more indications approved under the Orphan Drug Act of 1983. The Orphan Drug Act supports the development of innovative treatments for rare disease patients. The creation of the orphan drug designation with the passage of the Orphan Drug Act in 1983 has facilitated the development and approval of drugs for rare diseases and 2017 and 2018, were marked by the highest number of orphan drug and indication approvals to date. Production of gene therapies is associated with use of high-end technologies, high research and development costs, and skilled scientists and researchers, which reflects in high prices of these therapies.

Market Dynamics

Key players in the market are dedicated to the development of gene therapies for the novel indications such as Huntington's disease for which there are no specific approved medication. For instance, uniQure N.V. is developing AMT-130, a novel Gene therapy for Huntington's disease (HD), a rare, fatal, neurodegenerative genetic disorder. AMT-130 is currently in pre-clinical trial and is expected to receive approval in near future. Furthermore, companies are developing novel gene therapies for rare diseases such as Hemophilia B and Hemophilia A, which can offer long term treatment and can replace lifelong medication for this diseases. Launches of such novel therapies in near future is expected to significantly create lucrative opportunity for growth of the market. Moreover, increasing reimbursement support as well as innovative payment options are being adopted by manufacturers such as pay for performance. However, high prices of these therapies and potential challenges in commercialization of these therapies in emerging economies such as Asia Pacific and Latin America may restrain growth of the market

Key features of the study

It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market
This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategy adopted by the leading players
It profiles leading players in the global Gene Therapy for Rare Disease Market based on the following parameters - company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies
Key companies covered as a part of this study include Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.
Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launches, by Technology up-gradation, market expansion, and marketing tactics
The global Gene Therapy for Rare Disease Market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
Stakeholders would have ease in decision making through the various strategy matrices used in analyzing the Gene Therapy for Rare Disease Market

Market Segmentation

By Drug (Revenue, USD Mn, 2021-2033)
Approved Drugs
- Tisagenlecleucel (Kymriah)
- Axicabtagene ciloleucel (Yescarta)
- Voretigene neparvovec (Luxturna)
- Strimvelis
Pipeline Drugs
- GT-AADC
- Fidanacogene elaparvovec (SPK-9011)
- OTL-200
- bb2121
- AMT-061
- Others
By Therapeutic Application (Revenue, USD Mn, 2021-2033)
Oncology
Neurological Disorders
Ophthalmic Disorders
Hematological Disorders
Immunodeficiency Disorders
Metabolic Disorders
Others
Global Gene Therapy for Rare Disease Market, By Region: (Revenue, USD Mn, 2021-2033)
North America
- U.S.
- Canada
Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
Europe
- Germany
- U.K.
- France
- Italy
- Spain
- Russia
- Rest of Europe
Asia Pacific
- China
- India
- Japan
- Australia
- South Korea
- ASEAN
- Rest of Asia Pacific
Middle East
- GCC
- Israel
- Rest of Middle East
Africa
- South Africa
- Central Africa
- North Africa
Key Players
Kite Pharma, Inc. (Gilead Sciences, Inc.)
Novartis International AG
Juno Therapeutics Inc. (Celgene Corporation)
Bluebird Bio, Inc.
Spark Therapeutics, Inc.
uniQure N.V.
Orchard Therapeutics Plc.
PTC Therapeutics, Inc.
BioMarin Pharmaceutical Inc.

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