Gene Therapy For Rare Disease Market Size and Forecasts (2021 - 2031), Global and Regional Share, Trends, and Growth Opportunity Analysis
Gene therapy is used to cures for many diseases and types of medical treatment. Many diseases such as, cancers, neurological disorders, cardiovascular disease, infectious diseases and many more rare diseases. Gene therapy encompasses the manipulation of genes to fight or prevent diseases. The therapy is introduces a good gene into a person who has a disease caused by a bad gene. Many of treatments are include, biosimilars, vaccines, complex generics, gene therapies, immuno-therapies and novel drugs.
MARKET DYNAMICS
The gene therapy for rare diseases market is anticipated to grow in the forecast period owing to driving factors such as, launches of novel gene therapies and rise in the number of drug approvals for treatment of various rare diseases. Moreover, increasing gene therapy innovations for cardiovascular and rare diseases treatment is anticipated to offer more growth opportunities for the players operating in the gene therapy for rare diseases market.
MARKET SCOPE
The "Global Gene Therapy for Rare Diseases Market Analysis to 2031" is a specialized and in-depth study of the pharmaceutical industry with a special focus on the global market trend analysis. The report aims to provide an overview of gene therapy for rare diseases market with detailed market segmentation by therapeutic application and geography. The global gene therapy for rare diseases market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading gene therapy for rare diseases market players and offers key trends and opportunities in the market.
MARKET SEGMENTATION
The global gene therapy for rare diseases market is segmented on the basis of therapeutic application. Based on therapeutic application, the market is segmented as, oncology, neurological disorders, ophthalmic disorders, hematological disorders, immunodeficiency disorders, metabolic disorders and others.
REGIONAL FRAMEWORK
The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global gene therapy for rare diseases market based on various segments. It also provides market size and forecast estimates from year 2021 to 2031 with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The gene therapy for rare diseases market by each region is later sub-segmented by respective countries and segments. The report covers analysis and forecast of 18 countries globally along with current trend and opportunities prevailing in the region.
The report analyzes factors affecting gene therapy for rare diseases market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the gene therapy for rare diseases market in these regions.
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The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global gene therapy for rare diseases market based on various segments. It also provides market size and forecast estimates from year 2021 to 2031 with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The gene therapy for rare diseases market by each region is later sub-segmented by respective countries and segments. The report covers analysis and forecast of 18 countries globally along with current trend and opportunities prevailing in the region.
The report analyzes factors affecting gene therapy for rare diseases market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the gene therapy for rare diseases market in these regions.
MARKET PLAYERS
The reports cover key developments in the gene therapy for rare diseases market as organic and inorganic growth strategies. Various companies are focusing on organic growth strategies such as product launches, product approvals and others such as patents and events. Inorganic growth strategies activities witnessed in the market were acquisitions and partnership & collaborations. These activities have paved way for expansion of business and customer base of market players. The market payers from gene therapy for rare diseases market are anticipated to lucrative growth opportunities in the future with the rising demand for gene therapy for rare diseases market in the global market. Below mentioned is the list of few companies engaged in the gene therapy for rare diseases market.
The report also includes the profiles of key gene therapy for rare diseases market companies along with their SWOT analysis and market strategies. In addition, the report focuses on leading industry players with information such as company profiles, components and services offered, financial information of last 3 years, key development in past five years.
• BioMarin
• Orchard Therapeutics plc.
• Spark Therapeutics, Inc.
• PTC Therapeutics
• bluebird bio, Inc.
• Kite Pharma
• uniQure N.V.
• Juno Therapeutics Inc. (Celgene Corporation)
• Novartis AG
• Other Company
The Insight Partner's dedicated research and analysis team consist of experienced professionals with advanced statistical expertise and offer various customization options in the existing study.
- Sample PDF showcases the content structure and the nature of the information with qualitative and quantitative analysis.
- Request discounts available for Start-Ups & Universities
Report Coverage
Revenue forecast, Company Analysis, Industry landscape, Growth factors, and Trends
Segment Covered
This text is related
to segments covered.
Regional Scope
North America, Europe, Asia Pacific, Middle East & Africa, South & Central America
Country Scope
This text is related
to country scope.
1. INTRODUCTION
1.1. SCOPE OF THE STUDY
1.2. THE INSIGHT PARTNERS RESEARCH REPORT GUIDANCE
1.3. MARKET SEGMENTATION
1.3.1 Gene Therapy For Rare Disease Market - By Therapeutic Application
1.3.2 Gene Therapy For Rare Disease Market - By Region
1.3.2.1 By Country
2. KEY TAKEWAYS
3. RESEARCH METHODOLOGY
4. GENE THERAPY FOR RARE DISEASE MARKET LANDSCAPE
4.1. OVERVIEW
4.2. PEST ANALYSIS
4.2.1 North America - Pest Analysis
4.2.2 Europe - Pest Analysis
4.2.3 Asia-Pacific - Pest Analysis
4.2.4 Middle East and Africa - Pest Analysis
4.2.5 South and Central America - Pest Analysis
4.3. EXPERT OPINIONS
5. GENE THERAPY FOR RARE DISEASE MARKET - KEY MARKET DYNAMICS
5.1. KEY MARKET DRIVERS
5.2. KEY MARKET RESTRAINTS
5.3. KEY MARKET OPPORTUNITIES
5.4. FUTURE TRENDS
5.5. IMPACT ANALYSIS OF DRIVERS, RESTRAINTS & EXPECTED INFLUENCE OF COVID-19 PANDEMIC
6. GENE THERAPY FOR RARE DISEASE MARKET - GLOBAL MARKET ANALYSIS
6.1. GENE THERAPY FOR RARE DISEASE - GLOBAL MARKET OVERVIEW
6.2. GENE THERAPY FOR RARE DISEASE - GLOBAL MARKET AND FORECAST TO 2028
6.3. MARKET POSITIONING/MARKET SHARE
7. GENE THERAPY FOR RARE DISEASE MARKET - REVENUE AND FORECASTS TO 2028 - THERAPEUTIC APPLICATION
7.1. OVERVIEW
7.2. THERAPEUTIC APPLICATION MARKET FORECASTS AND ANALYSIS
7.3. ONCOLOGY
7.3.1. Overview
7.3.2. Oncology Market Forecast and Analysis
7.4. NEUROLOGICAL DISORDERS
7.4.1. Overview
7.4.2. Neurological Disorders Market Forecast and Analysis
7.5. OPHTHALMIC DISORDERS
7.5.1. Overview
7.5.2. Ophthalmic Disorders Market Forecast and Analysis
7.6. HEMATOLOGICAL DISORDERS
7.6.1. Overview
7.6.2. Hematological Disorders Market Forecast and Analysis
7.7. IMMUNODEFICIENCY DISORDERS
7.7.1. Overview
7.7.2. Immunodeficiency Disorders Market Forecast and Analysis
7.8. METABOLIC DISORDERS
7.8.1. Overview
7.8.2. Metabolic Disorders Market Forecast and Analysis
7.9. OTHERS
7.9.1. Overview
7.9.2. Others Market Forecast and Analysis
8. GENE THERAPY FOR RARE DISEASE MARKET REVENUE AND FORECASTS TO 2028 - GEOGRAPHICAL ANALYSIS
8.1. NORTH AMERICA
8.1.1 North America Gene Therapy For Rare Disease Market Overview
8.1.2 North America Gene Therapy For Rare Disease Market Forecasts and Analysis
8.1.3 North America Gene Therapy For Rare Disease Market Forecasts and Analysis - By Therapeutic Application
8.1.4 North America Gene Therapy For Rare Disease Market Forecasts and Analysis - By Countries
8.1.4.1 United States Gene Therapy For Rare Disease Market
8.1.4.1.1 United States Gene Therapy For Rare Disease Market by Therapeutic Application
8.1.4.2 Canada Gene Therapy For Rare Disease Market
8.1.4.2.1 Canada Gene Therapy For Rare Disease Market by Therapeutic Application
8.1.4.3 Mexico Gene Therapy For Rare Disease Market
8.1.4.3.1 Mexico Gene Therapy For Rare Disease Market by Therapeutic Application
8.2. EUROPE
8.2.1 Europe Gene Therapy For Rare Disease Market Overview
8.2.2 Europe Gene Therapy For Rare Disease Market Forecasts and Analysis
8.2.3 Europe Gene Therapy For Rare Disease Market Forecasts and Analysis - By Therapeutic Application
8.2.4 Europe Gene Therapy For Rare Disease Market Forecasts and Analysis - By Countries
8.2.4.1 Germany Gene Therapy For Rare Disease Market
8.2.4.1.1 Germany Gene Therapy For Rare Disease Market by Therapeutic Application
8.2.4.2 France Gene Therapy For Rare Disease Market
8.2.4.2.1 France Gene Therapy For Rare Disease Market by Therapeutic Application
8.2.4.3 Italy Gene Therapy For Rare Disease Market
8.2.4.3.1 Italy Gene Therapy For Rare Disease Market by Therapeutic Application
8.2.4.4 Spain Gene Therapy For Rare Disease Market
8.2.4.4.1 Spain Gene Therapy For Rare Disease Market by Therapeutic Application
8.2.4.5 United Kingdom Gene Therapy For Rare Disease Market
8.2.4.5.1 United Kingdom Gene Therapy For Rare Disease Market by Therapeutic Application
8.3. ASIA-PACIFIC
8.3.1 Asia-Pacific Gene Therapy For Rare Disease Market Overview
8.3.2 Asia-Pacific Gene Therapy For Rare Disease Market Forecasts and Analysis
8.3.3 Asia-Pacific Gene Therapy For Rare Disease Market Forecasts and Analysis - By Therapeutic Application
8.3.4 Asia-Pacific Gene Therapy For Rare Disease Market Forecasts and Analysis - By Countries
8.3.4.1 Australia Gene Therapy For Rare Disease Market
8.3.4.1.1 Australia Gene Therapy For Rare Disease Market by Therapeutic Application
8.3.4.2 China Gene Therapy For Rare Disease Market
8.3.4.2.1 China Gene Therapy For Rare Disease Market by Therapeutic Application
8.3.4.3 India Gene Therapy For Rare Disease Market
8.3.4.3.1 India Gene Therapy For Rare Disease Market by Therapeutic Application
8.3.4.4 South Korea Gene Therapy For Rare Disease Market
8.3.4.4.1 South Korea Gene Therapy For Rare Disease Market by Therapeutic Application
8.3.4.5 Indonesia Gene Therapy For Rare Disease Market
8.3.4.5.1 Indonesia Gene Therapy For Rare Disease Market by Therapeutic Application
8.4. MIDDLE EAST AND AFRICA
8.4.1 Middle East and Africa Gene Therapy For Rare Disease Market Overview
8.4.2 Middle East and Africa Gene Therapy For Rare Disease Market Forecasts and Analysis
8.4.3 Middle East and Africa Gene Therapy For Rare Disease Market Forecasts and Analysis - By Therapeutic Application
8.4.4 Middle East and Africa Gene Therapy For Rare Disease Market Forecasts and Analysis - By Countries
8.4.4.1 South Africa Gene Therapy For Rare Disease Market
8.4.4.1.1 South Africa Gene Therapy For Rare Disease Market by Therapeutic Application
8.4.4.2 Saudi Arabia Gene Therapy For Rare Disease Market
8.4.4.2.1 Saudi Arabia Gene Therapy For Rare Disease Market by Therapeutic Application
8.4.4.3 U.A.E Gene Therapy For Rare Disease Market
8.4.4.3.1 U.A.E Gene Therapy For Rare Disease Market by Therapeutic Application
8.5. SOUTH AND CENTRAL AMERICA
8.5.1 South and Central America Gene Therapy For Rare Disease Market Overview
8.5.2 South and Central America Gene Therapy For Rare Disease Market Forecasts and Analysis
8.5.3 South and Central America Gene Therapy For Rare Disease Market Forecasts and Analysis - By Therapeutic Application
8.5.4 South and Central America Gene Therapy For Rare Disease Market Forecasts and Analysis - By Countries
8.5.4.1 Brazil Gene Therapy For Rare Disease Market
8.5.4.1.1 Brazil Gene Therapy For Rare Disease Market by Therapeutic Application
8.5.4.2 Argentina Gene Therapy For Rare Disease Market
8.5.4.2.1 Argentina Gene Therapy For Rare Disease Market by Therapeutic Application
9. INDUSTRY LANDSCAPE
9.1. MERGERS AND ACQUISITIONS
9.2. AGREEMENTS, COLLABORATIONS AND JOIN VENTURES
9.3. NEW PRODUCT LAUNCHES
9.4. EXPANSIONS AND OTHER STRATEGIC DEVELOPMENTS
10. GENE THERAPY FOR RARE DISEASE MARKET, KEY COMPANY PROFILES
10.1. KITE PHARMA, INC. (GILEAD SCIENCES, INC.)
10.1.1. Key Facts
10.1.2. Business Description
10.1.3. Products and Services
10.1.4. Financial Overview
10.1.5. SWOT Analysis
10.1.6. Key Developments
10.2. NOVARTIS AG
10.2.1. Key Facts
10.2.2. Business Description
10.2.3. Products and Services
10.2.4. Financial Overview
10.2.5. SWOT Analysis
10.2.6. Key Developments
10.3. JUNO THERAPEUTICS INC. (CELGENE CORPORATION)
10.3.1. Key Facts
10.3.2. Business Description
10.3.3. Products and Services
10.3.4. Financial Overview
10.3.5. SWOT Analysis
10.3.6. Key Developments
10.4. BLUEBIRD BIO, INC.
10.4.1. Key Facts
10.4.2. Business Description
10.4.3. Products and Services
10.4.4. Financial Overview
10.4.5. SWOT Analysis
10.4.6. Key Developments
10.5. SPARK THERAPEUTICS, INC.
10.5.1. Key Facts
10.5.2. Business Description
10.5.3. Products and Services
10.5.4. Financial Overview
10.5.5. SWOT Analysis
10.5.6. Key Developments
10.6. UNIQURE N.V
10.6.1. Key Facts
10.6.2. Business Description
10.6.3. Products and Services
10.6.4. Financial Overview
10.6.5. SWOT Analysis
10.6.6. Key Developments
10.7. ORCHARD THERAPEUTICS PLC.
10.7.1. Key Facts
10.7.2. Business Description
10.7.3. Products and Services
10.7.4. Financial Overview
10.7.5. SWOT Analysis
10.7.6. Key Developments
10.8. PTC THERAPEUTICS INC.
10.8.1. Key Facts
10.8.2. Business Description
10.8.3. Products and Services
10.8.4. Financial Overview
10.8.5. SWOT Analysis
10.8.6. Key Developments
10.9. BIOMARIN PHARMACEUTICAL INC.
10.9.1. Key Facts
10.9.2. Business Description
10.9.3. Products and Services
10.9.4. Financial Overview
10.9.5. SWOT Analysis
10.9.6. Key Developments
10.10. OTHER COMPANY
10.10.1. Key Facts
10.10.2. Business Description
10.10.3. Products and Services
10.10.4. Financial Overview
10.10.5. SWOT Analysis
10.10.6. Key Developments
11. APPENDIX
11.1. ABOUT THE INSIGHT PARTNERS
11.2. GLOSSARY OF TERMS
1. BioMarin
2. Orchard Therapeutics plc.
3. Spark Therapeutics, Inc.
4. PTC Therapeutics
5. bluebird bio, Inc.
6. Kite Pharma
7. uniQure N.V.
8. Juno Therapeutics Inc. (Celgene Corporation)
9. Novartis AG
10. Other Company
The Insight Partners performs research in 4 major stages: Data Collection & Secondary Research, Primary Research, Data Analysis and Data Triangulation & Final Review.
- Data Collection and Secondary Research:
As a market research and consulting firm operating from a decade, we have published many reports and advised several clients across the globe. First step for any study will start with an assessment of currently available data and insights from existing reports. Further, historical and current market information is collected from Investor Presentations, Annual Reports, SEC Filings, etc., and other information related to company’s performance and market positioning are gathered from Paid Databases (Factiva, Hoovers, and Reuters) and various other publications available in public domain.
Several associations trade associates, technical forums, institutes, societies and organizations are accessed to gain technical as well as market related insights through their publications such as research papers, blogs and press releases related to the studies are referred to get cues about the market. Further, white papers, journals, magazines, and other news articles published in the last 3 years are scrutinized and analyzed to understand the current market trends.
- Primary Research:
The primarily interview analysis comprise of data obtained from industry participants interview and answers to survey questions gathered by in-house primary team.
For primary research, interviews are conducted with industry experts/CEOs/Marketing Managers/Sales Managers/VPs/Subject Matter Experts from both demand and supply side to get a 360-degree view of the market. The primary team conducts several interviews based on the complexity of the markets to understand the various market trends and dynamics which makes research more credible and precise.
A typical research interview fulfils the following functions:
- Provides first-hand information on the market size, market trends, growth trends, competitive landscape, and outlook
- Validates and strengthens in-house secondary research findings
- Develops the analysis team’s expertise and market understanding
Primary research involves email interactions and telephone interviews for each market, category, segment, and sub-segment across geographies. The participants who typically take part in such a process include, but are not limited to:
- Industry participants: VPs, business development managers, market intelligence managers and national sales managers
- Outside experts: Valuation experts, research analysts and key opinion leaders specializing in the electronics and semiconductor industry.
Below is the breakup of our primary respondents by company, designation, and region:
Once we receive the confirmation from primary research sources or primary respondents, we finalize the base year market estimation and forecast the data as per the macroeconomic and microeconomic factors assessed during data collection.
- Data Analysis:
Once data is validated through both secondary as well as primary respondents, we finalize the market estimations by hypothesis formulation and factor analysis at regional and country level.
- 3.1 Macro-Economic Factor Analysis:
We analyse macroeconomic indicators such the gross domestic product (GDP), increase in the demand for goods and services across industries, technological advancement, regional economic growth, governmental policies, the influence of COVID-19, PEST analysis, and other aspects. This analysis aids in setting benchmarks for various nations/regions and approximating market splits. Additionally, the general trend of the aforementioned components aid in determining the market's development possibilities.
- 3.2 Country Level Data:
Various factors that are especially aligned to the country are taken into account to determine the market size for a certain area and country, including the presence of vendors, such as headquarters and offices, the country's GDP, demand patterns, and industry growth. To comprehend the market dynamics for the nation, a number of growth variables, inhibitors, application areas, and current market trends are researched. The aforementioned elements aid in determining the country's overall market's growth potential.
- 3.3 Company Profile:
The “Table of Contents” is formulated by listing and analyzing more than 25 - 30 companies operating in the market ecosystem across geographies. However, we profile only 10 companies as a standard practice in our syndicate reports. These 10 companies comprise leading, emerging, and regional players. Nonetheless, our analysis is not restricted to the 10 listed companies, we also analyze other companies present in the market to develop a holistic view and understand the prevailing trends. The “Company Profiles” section in the report covers key facts, business description, products & services, financial information, SWOT analysis, and key developments. The financial information presented is extracted from the annual reports and official documents of the publicly listed companies. Upon collecting the information for the sections of respective companies, we verify them via various primary sources and then compile the data in respective company profiles. The company level information helps us in deriving the base number as well as in forecasting the market size.
- 3.4 Developing Base Number:
Aggregation of sales statistics (2020-2022) and macro-economic factor, and other secondary and primary research insights are utilized to arrive at base number and related market shares for 2022. The data gaps are identified in this step and relevant market data is analyzed, collected from paid primary interviews or databases. On finalizing the base year market size, forecasts are developed on the basis of macro-economic, industry and market growth factors and company level analysis.
- Data Triangulation and Final Review:
The market findings and base year market size calculations are validated from supply as well as demand side. Demand side validations are based on macro-economic factor analysis and benchmarks for respective regions and countries. In case of supply side validations, revenues of major companies are estimated (in case not available) based on industry benchmark, approximate number of employees, product portfolio, and primary interviews revenues are gathered. Further revenue from target product/service segment is assessed to avoid overshooting of market statistics. In case of heavy deviations between supply and demand side values, all thes steps are repeated to achieve synchronization.
We follow an iterative model, wherein we share our research findings with Subject Matter Experts (SME’s) and Key Opinion Leaders (KOLs) until consensus view of the market is not formulated – this model negates any drastic deviation in the opinions of experts. Only validated and universally acceptable research findings are quoted in our reports.
We have important check points that we use to validate our research findings – which we call – data triangulation, where we validate the information, we generate from secondary sources with primary interviews and then we re-validate with our internal data bases and Subject matter experts. This comprehensive model enables us to deliver high quality, reliable data in shortest possible time.
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