Gene Therapy For Rare Disease Market 2027 by Types, Application, Technology, Opportunities, End Users and Regions | The Insight Partners

Gene Therapy For Rare Disease Market to 2027 - Global Analysis and Forecasts By Therapeutic Application ( Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, Others )

Report Code: TIPRE00003413 | No. of Pages: 150 | Category: Pharmaceuticals | Status: Upcoming
MARKET INTRODUCTION
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 2027" 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 2017 to 2027 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.
TABLE OF CONTENTS

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 AND RESTRAINTS

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 2027
6.3. MARKET POSITIONING/MARKET SHARE

7. GENE THERAPY FOR RARE DISEASE MARKET - REVENUE AND FORECASTS TO 2027 - 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 2027 - 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
The List of Companies

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