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시장보고서
상품코드
2084681
희귀질환용 유전자 치료 시장 : 약물별, 치료 용도별, 지역별Gene Therapy for Rare Disease Market, By Drug, By Therapeutic Application, By Geography |
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희귀질환용 유전자 치료 시장은 2026년에 2억 4,250만 달러 규모가 될 것으로 추정되며, 2033년까지 19억 7,870만 달러에 달할 것으로 예상됩니다. 2026년부터 2033년까지 CAGR 35%로 성장할 것으로 전망됩니다.
| 분석 범위 | 분석 상세 | ||
|---|---|---|---|
| 기준 연도 | 2025년 | 시장 규모(2026년) | 2억 4,250만 달러 |
| 실적 데이터 | 2020-2024년 | 예측 기간 | 2026-2033년 |
| 예측 기간 CAGR(2026-2033년) | 35.00% | 예측 금액(2033년) | 19억 7,870만 달러 |
최근 몇 년간, 유전자 치료의 상당수가 희귀질환 치료제(오펀 드럭)로 지정되고 있습니다. 희귀질환 치료제는 일반적으로 1983년 제정된 ‘희귀질환 치료제법(Orphan Drug Act)’에 근거하여, 하나 이상의 적응증에 대해 승인된 의약품으로 정의됩니다. 이 법은 희귀질환 환자를 위한 혁신적인 치료법 개발을 지원하기 위한 것입니다. 1983년 희귀질환 의약품법의 제정에 따라 희귀질환 의약품 지정 제도가 신설되면서, 희귀질환 치료제 개발 및 승인이 촉진되었으며, 2017년과 2018년에는 사상 최다 규모의 희귀질환 의약품 및 적응증 승인이 이루어졌습니다. 유전자 치료제 제조에는 최첨단 기술의 활용, 막대한 연구개발비, 그리고 숙련된 과학자와 연구원의 참여가 필요하며, 이러한 요소들이 이 치료법들의 높은 가격에 반영되어 있습니다.
시장 역학
시장의 주요 기업들은 특정 승인된 치료제가 없는 헌팅턴병 등 새로운 적응증에 대한 유전자 치료법 개발에 주력하고 있습니다. 예를 들어, uniQure N.V.는 희귀하고 치명적인 신경퇴행성 유전 질환인 헌팅턴병(HD)에 대한 새로운 유전자 치료제 'AMT-130'을 개발하고 있습니다. AMT-130은 현재 전임상시험 단계에 있으며, 가까운 시일 내에 승인을 받을 것으로 예상됩니다. 또한, 각 기업은 B형 혈우병이나 A형 혈우병과 같은 희귀 질환에 대한 새로운 유전자 치료법을 개발하고 있으며, 이러한 치료법은 장기적인 치료를 가능하게 하고, 해당 질환에 대한 평생 약물 치료를 대체할 가능성이 있습니다. 가까운 시일 내에 이러한 새로운 치료법이 시장에 출시됨에 따라, 시장 성장을 위한 매우 유망한 기회가 대폭 창출될 것으로 예상됩니다. 또한, 상환 지원 확대와 더불어 각 제조사들은 ‘성과 연동형 지불(pay for performance)’ 등의 혁신적인 지불 방식을 도입하고 있습니다. 그러나 이러한 치료법의 높은 가격과 아시아태평양 및 라틴아메리카 등 신흥 경제권에서의 상용화에 따른 잠재적 과제가 시장 성장을 저해할 가능성이 있습니다.
본 보고서의 주요 특징
- 본 보고서는 각 부문의 잠재적 수익 기회를 밝히고, 이 시장의 매력적인 투자 제안 매트릭스에 대해 설명하고 있습니다.
- 또한, 시장의 촉진요인 및 억제요인, 기회, 신제품 출시 및 승인, 시장 동향, 지역별 전망, 주요 기업들이 채택하는 경쟁 전략 등에 관한 중요한 인사이트도 제공하고 있습니다.
- 전 세계 희귀질환용 유전자 치료 시장의 주요 기업 개요을 각종 지표(기업 개요, 제품 포트폴리오, 주요 동향, 재무 실적, 전략 등)를 바탕으로 수록하고 있습니다.
- 본 보고서의 인사이트를 바탕으로, 마케팅 담당자와 기업 경영진은 향후 제품 출시, 기술 업데이트, 시장 확대 및 마케팅 전략에 대해 충분한 정보를 바탕으로 의사결정을 내릴 수 있습니다.
- '세계 희귀질환용 유전자 치료 시장' 보고서는 투자자, 공급업체, 제품 제조업체, 유통업체, 신규 진입 기업, 재무 분석가 등 이 업계의 다양한 이해관계자들을 대상으로 합니다.
- 이해관계자들은 전 세계 희귀질환용 유전자 치료 시장 분석에 사용되는 다양한 전략 매트릭스를 통해 의사결정을 보다 수월하게 할 수 있습니다.
목차
제1장 분석 목적과 가정
제2장 시장 전망
제3장 시장 역학·규제·동향 분석
제4장 세계의 희귀질환용 유전자 치료 시장 : 약물별(2021-2033년)
제5장 세계의 희귀질환용 유전자 치료 시장 : 치료 용도별(2021-2033년)
제6장 세계의 희귀질환용 유전자 치료 시장 : 지역별(2021-2033년)
제7장 경쟁 구도
제8장 섹션
KSM 26.07.16Gene 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.
Table of Contents
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
- Kite Pharma, Inc. (Gilead Sciences, Inc.)
- Analyst Views
8. Section
- References
- Research Methodology
- About Us
