Gene Therapy for Rare Disease Market
- 2026 年市場規模
- USD 242.5 Mn
- 2033 年預估規模
- USD 1,978.7 Mn
- 年複合成長率 CAGR
- 35%
以上數字由 Coherent Market Insights 於本報告中提出，量子訊息原樣引用。
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完整報告名稱與涵蓋範圍
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)
報告摘要
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
• This report provides in-depth analysis of gene therapy for rare disease market and provides market size (US$ Million) and Cumulative Annual Growth Rate (CAGR %) for the forecast period (2026 - 2033), considering 2025, as the base year
• 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)
o Approved Drugs
Tisagenlecleucel (Kymriah)
Axicabtagene ciloleucel (Yescarta)
Voretigene neparvovec (Luxturna)
Strimvelis
o Pipeline Drugs
GT-AADC
Fidanacogene elaparvovec (SPK-9011)
OTL-200
bb2121
AMT-061
Others
• By Therapeutic Application (Revenue, USD Mn, 2021-2033)
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
• Global Gene Therapy for Rare Disease Market, By Region: (Revenue, USD Mn, 2021-2033)
o North America
U.S.
Canada
o Latin America
Brazil
Mexico
Argentina
Rest of Latin America
o Europe
Germany
U.K.
France
Italy
Spain
Russia
Rest of Europe
o Asia Pacific
China
India
Japan
Australia
South Korea
ASEAN
Rest of Asia Pacific
o Middle East
GCC
Israel
Rest of Middle East
o Africa
South Africa
Central Africa
North Africa
• Key Players
o Kite Pharma, Inc. (Gilead Sciences, Inc.)
o Novartis International AG
o Juno Therapeutics Inc. (Celgene Corporation)
o Bluebird Bio, Inc.
o Spark Therapeutics, Inc.
o uniQure N.V.
o Orchard Therapeutics Plc.
o PTC Therapeutics, Inc.
o BioMarin Pharmaceutical Inc.
目錄 Table of Contents
Table of Contents
1. Research Objectives and Assumptions
o Research Objectives
o Assumptions
o Abbreviations
2. Market Purview
o Report Description
Market Definition and Scope
o Executive Summary
Market Snapshot, By Drug
Market Snapshot, By Therapeutic Application
Market Snapshot, By Region
o Coherent Opportunity Map (COM)
3. Market Dynamics, Regulations, and Trends Analysis
o Market Dynamics
Drivers
Restraints
Market Opportunities
o Impact Analysis
o Market Trends
o Key Developments
o Product Approval
o Collaboration/Merge/Acquisition
o Regulatory Landscape
o PEST Analysis
o Porter’s Five Forces Analysis
o Epidemiology
o Robust Pipeline Analysis
4. Global Gene Therapy for Rare Disease Market, By Drug, 2021 – 2033, (USD Mn)
o Introduction
Market Share Analysis, 2026 and 2033 (%)
Y-o-Y Growth Analysis, 2022– 2033
Segment Trends
o Approved Drugs
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
5. Global Gene Therapy for Rare Disease Market, By Therapeutic Application, 2021 – 2033, (USD Mn)
o Introduction
Market Share Analysis, 2026 and 2033 (%)
Y-o-Y Growth Analysis, 2022 – 2033
Segment Trends
o Oncology
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o Neurological Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o Ophthalmic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o Hematological Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o Immunodeficiency Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o Metabolic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
o 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)
o Introduction
Market Share Analysis, By Region, 2026 and 2033 (%)
Y-o-Y Growth Analysis, For Regions, 2022–2033
Regional Trends
o 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)
o U.S.
o Canada
o 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)
o Brazil
o Mexico
o Argentina
o Rest of Latin America
o 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)
o U.K.
o Germany
o Italy
o France
o Spain
o Russia
o Rest of Europe
o 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)
o China
o India
o Japan
o ASEAN
o Australia
o South Korea
o Rest of Asia Pacific
o 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)
o GCC
o Israel
o Rest of Middle East
o 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)
o North Africa
o Central Africa
o South Africa
7. Competitive Landscape
o Heat Map Analysis
o Market Share Analysis
o Company Profile
Kite Pharma, Inc. (Gilead Sciences, Inc.)
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
Novartis International AG
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
Juno Therapeutics Inc. (Celgene Corporation)
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
Bluebird Bio, Inc.
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
Spark Therapeutics, Inc.
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
uniQure N.V.
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
Orchard Therapeutics Plc.
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
PTC Therapeutics, Inc.
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
BioMarin Pharmaceutical Inc.
o Company Highlights
o Product Portfolio
o Financial Performance
o Recent Highlights
o Market Strategies
o Analyst Views
8. Section
o References
o Research Methodology
o About Us
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