Market Overview:
Intravenous is expected to be the largest segment due to its high adoption rate and easy availability. Among these applications, Mucpolysaccarhoidos III A is expected to be the fastest growing segment due its high incidence rate and increasing awareness about this disease among patients and healthcare professionals.
Product Definition:
Gene therapy is the therapeutic application of nucleic acids (DNA or RNA) to a patient's cells to treat disease. It can be used to add a gene, replace a defective gene, or inactivate a harmful gene.
Intravenous:
Intravenous (IV) is a route of drug delivery which involves injecting the drug into the blood vessels. The IV formulation is one of the most preferred methods for delivering drugs due to its several advantages such as it does not require oral intake and can be administered at constant dosage. Intravenous administration also allows for higher bioavailability since it bypasses first pass metabolism in liver, thereby allowing more amount of active ingredient to reach systemic circulation.
ICV:
ICV is an enzyme that helps in the processing of glycosaminoglycans (GAGs) and other polysaccharides. The deficiency of ICV leads to a buildup of mucopolysaccharides and hyaluronic acid in various tissues, including eyes, brain, skin, liver & gut. MPSS is caused by mutations in the PGM1 gene which codes for ICV.
Application Insights:
The other types such as MPS IIIA.
Regional Analysis:
North America dominated the global market in 2017. This can be attributed to the presence of a large number of patients’ association, research institutes and companies involved in developing novel therapies for MPS disorders. Moreover, an increase in funding from public & private entities is anticipated to boost growth during the forecast period. For instance, since 2012 till 2016; National Institute of Neurological Disorders & Strokes (NINDS) invested around USD X million to fund 13 gene therapy clinical trials for Niemann-Pick Type C disease (NP-C).
Asia Pacific is expected to witness lucrative growth over the forecast period owing to increasing awareness about these diseases and their treatment options among people affected with MPS disorders as well as their families & caretakers.
Growth Factors:
- Increasing prevalence of Mucopolysaccharidosis
- Rising awareness about Gene Therapy for Mucopolysaccharidosis
- Technological advancements in the field of gene therapy
- Growing number of clinical trials for Gene Therapy for Mucopolysaccharidosis
- increasing investment in research and development
Scope Of The Report
Report Attributes
Report Details
Report Title
Gene Therapy for Mucopolysaccharidosis Market Research Report
By Type
Intravenous, ICV, Intracerebral, Intracisternal
By Application
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III A, Mucopolysaccharidosis III B
By Companies
Sangamo Therapeutics, Swedish Orphan Biovitrum, uniQure
Regions Covered
North America, Europe, APAC, Latin America, MEA
Base Year
2021
Historical Year
2019 to 2020 (Data from 2010 can be provided as per availability)
Forecast Year
2030
Number of Pages
204
Number of Tables & Figures
143
Customization Available
Yes, the report can be customized as per your need.
Global Gene Therapy for Mucopolysaccharidosis Market Report Segments:
The global Gene Therapy for Mucopolysaccharidosis market is segmented on the basis of:
Types
Intravenous, ICV, Intracerebral, Intracisternal
The product segment provides information about the market share of each product and the respective CAGR during the forecast period. It lays out information about the product pricing parameters, trends, and profits that provides in-depth insights of the market. Furthermore, it discusses latest product developments & innovation in the market.
Applications
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III A, Mucopolysaccharidosis III B
The application segment fragments various applications of the product and provides information on the market share and growth rate of each application segment. It discusses the potential future applications of the products and driving and restraining factors of each application segment.
Some of the companies that are profiled in this report are:
- Sangamo Therapeutics
- Swedish Orphan Biovitrum
- uniQure
Highlights of The Gene Therapy for Mucopolysaccharidosis Market Report:
- The market structure and projections for the coming years.
- Drivers, restraints, opportunities, and current trends of market.
- Historical data and forecast.
- Estimations for the forecast period 2030.
- Developments and trends in the market.
- By Type:
- Intravenous
- ICV
- Intracerebral
- Intracisternal
- By Application:
- Mucopolysaccharidosis I
- Mucopolysaccharidosis II
- Mucopolysaccharidosis III A
- Mucopolysaccharidosis III B
- Market scenario by region, sub-region, and country.
- Market share of the market players, company profiles, product specifications, SWOT analysis, and competitive landscape.
- Analysis regarding upstream raw materials, downstream demand, and current market dynamics.
- Government Policies, Macro & Micro economic factors are also included in the report.
We have studied the Gene Therapy for Mucopolysaccharidosis Market in 360 degrees via. both primary & secondary research methodologies. This helped us in building an understanding of the current market dynamics, supply-demand gap, pricing trends, product preferences, consumer patterns & so on. The findings were further validated through primary research with industry experts & opinion leaders across countries. The data is further compiled & validated through various market estimation & data validation methodologies. Further, we also have our in-house data forecasting model to predict market growth up to 2030.
Regional Analysis
- North America
- Europe
- Asia Pacific
- Middle East & Africa
- Latin America
Note: A country of choice can be added in the report at no extra cost. If more than one country needs to be added, the research quote will vary accordingly.
The geographical analysis part of the report provides information about the product sales in terms of volume and revenue in regions. It lays out potential opportunities for the new entrants, emerging players, and major players in the region. The regional analysis is done after considering the socio-economic factors and government regulations of the countries in the regions.
How you may use our products:
- Correctly Positioning New Products
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8 Reasons to Buy This Report
- Includes a Chapter on the Impact of COVID-19 Pandemic On the Market
- Report Prepared After Conducting Interviews with Industry Experts & Top Designates of the Companies in the Market
- Implemented Robust Methodology to Prepare the Report
- Includes Graphs, Statistics, Flowcharts, and Infographics to Save Time
- Industry Growth Insights Provides 24/5 Assistance Regarding the Doubts in the Report
- Provides Information About the Top-winning Strategies Implemented by Industry Players.
- In-depth Insights On the Market Drivers, Restraints, Opportunities, and Threats
- Customization of the Report Available
Frequently Asked Questions?
Gene therapy is a treatment that uses genetic material to help cure or improve a disease. In mucopolysaccharidosis, gene therapy helps to repair the damage caused by the disease in the body's cells. This can help to improve symptoms and quality of life for people with this condition.
Some of the major companies in the gene therapy for mucopolysaccharidosis market are Sangamo Therapeutics, Swedish Orphan Biovitrum, uniQure.
Chapter 1 Executive Summary
Chapter 2 Assumptions and Acronyms Used
Chapter 3 Research Methodology
Chapter 4 Gene Therapy for Mucopolysaccharidosis Market Overview
4.1 Introduction
4.1.1 Market Taxonomy
4.1.2 Market Definition
4.1.3 Macro-Economic Factors Impacting the Market Growth
4.2 Gene Therapy for Mucopolysaccharidosis Market Dynamics
4.2.1 Market Drivers
4.2.2 Market Restraints
4.2.3 Market Opportunity
4.3 Gene Therapy for Mucopolysaccharidosis Market - Supply Chain Analysis
4.3.1 List of Key Suppliers
4.3.2 List of Key Distributors
4.3.3 List of Key Consumers
4.4 Key Forces Shaping the Gene Therapy for Mucopolysaccharidosis Market
4.4.1 Bargaining Power of Suppliers
4.4.2 Bargaining Power of Buyers
4.4.3 Threat of Substitution
4.4.4 Threat of New Entrants
4.4.5 Competitive Rivalry
4.5 Global Gene Therapy for Mucopolysaccharidosis Market Size & Forecast, 2018-2028
4.5.1 Gene Therapy for Mucopolysaccharidosis Market Size and Y-o-Y Growth
4.5.2 Gene Therapy for Mucopolysaccharidosis Market Absolute $ Opportunity
Chapter 5 Global Gene Therapy for Mucopolysaccharidosis Market Analysis and Forecast by Type
5.1 Introduction
5.1.1 Key Market Trends & Growth Opportunities by Type
5.1.2 Basis Point Share (BPS) Analysis by Type
5.1.3 Absolute $ Opportunity Assessment by Type
5.2 Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Type
5.2.1 Intravenous
5.2.2 ICV
5.2.3 Intracerebral
5.2.4 Intracisternal
5.3 Market Attractiveness Analysis by Type
Chapter 6 Global Gene Therapy for Mucopolysaccharidosis Market Analysis and Forecast by Applications
6.1 Introduction
6.1.1 Key Market Trends & Growth Opportunities by Applications
6.1.2 Basis Point Share (BPS) Analysis by Applications
6.1.3 Absolute $ Opportunity Assessment by Applications
6.2 Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Applications
6.2.1 Mucopolysaccharidosis I
6.2.2 Mucopolysaccharidosis II
6.2.3 Mucopolysaccharidosis III A
6.2.4 Mucopolysaccharidosis III B
6.3 Market Attractiveness Analysis by Applications
Chapter 7 Global Gene Therapy for Mucopolysaccharidosis Market Analysis and Forecast by Region
7.1 Introduction
7.1.1 Key Market Trends & Growth Opportunities by Region
7.1.2 Basis Point Share (BPS) Analysis by Region
7.1.3 Absolute $ Opportunity Assessment by Region
7.2 Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Region
7.2.1 North America
7.2.2 Europe
7.2.3 Asia Pacific
7.2.4 Latin America
7.2.5 Middle East & Africa (MEA)
7.3 Market Attractiveness Analysis by Region
Chapter 8 Coronavirus Disease (COVID-19) Impact
8.1 Introduction
8.2 Current & Future Impact Analysis
8.3 Economic Impact Analysis
8.4 Government Policies
8.5 Investment Scenario
Chapter 9 North America Gene Therapy for Mucopolysaccharidosis Analysis and Forecast
9.1 Introduction
9.2 North America Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Country
9.2.1 U.S.
9.2.2 Canada
9.3 Basis Point Share (BPS) Analysis by Country
9.4 Absolute $ Opportunity Assessment by Country
9.5 Market Attractiveness Analysis by Country
9.6 North America Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Type
9.6.1 Intravenous
9.6.2 ICV
9.6.3 Intracerebral
9.6.4 Intracisternal
9.7 Basis Point Share (BPS) Analysis by Type
9.8 Absolute $ Opportunity Assessment by Type
9.9 Market Attractiveness Analysis by Type
9.10 North America Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Applications
9.10.1 Mucopolysaccharidosis I
9.10.2 Mucopolysaccharidosis II
9.10.3 Mucopolysaccharidosis III A
9.10.4 Mucopolysaccharidosis III B
9.11 Basis Point Share (BPS) Analysis by Applications
9.12 Absolute $ Opportunity Assessment by Applications
9.13 Market Attractiveness Analysis by Applications
Chapter 10 Europe Gene Therapy for Mucopolysaccharidosis Analysis and Forecast
10.1 Introduction
10.2 Europe Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Country
10.2.1 Germany
10.2.2 France
10.2.3 Italy
10.2.4 U.K.
10.2.5 Spain
10.2.6 Russia
10.2.7 Rest of Europe
10.3 Basis Point Share (BPS) Analysis by Country
10.4 Absolute $ Opportunity Assessment by Country
10.5 Market Attractiveness Analysis by Country
10.6 Europe Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Type
10.6.1 Intravenous
10.6.2 ICV
10.6.3 Intracerebral
10.6.4 Intracisternal
10.7 Basis Point Share (BPS) Analysis by Type
10.8 Absolute $ Opportunity Assessment by Type
10.9 Market Attractiveness Analysis by Type
10.10 Europe Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Applications
10.10.1 Mucopolysaccharidosis I
10.10.2 Mucopolysaccharidosis II
10.10.3 Mucopolysaccharidosis III A
10.10.4 Mucopolysaccharidosis III B
10.11 Basis Point Share (BPS) Analysis by Applications
10.12 Absolute $ Opportunity Assessment by Applications
10.13 Market Attractiveness Analysis by Applications
Chapter 11 Asia Pacific Gene Therapy for Mucopolysaccharidosis Analysis and Forecast
11.1 Introduction
11.2 Asia Pacific Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Country
11.2.1 China
11.2.2 Japan
11.2.3 South Korea
11.2.4 India
11.2.5 Australia
11.2.6 South East Asia (SEA)
11.2.7 Rest of Asia Pacific (APAC)
11.3 Basis Point Share (BPS) Analysis by Country
11.4 Absolute $ Opportunity Assessment by Country
11.5 Market Attractiveness Analysis by Country
11.6 Asia Pacific Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Type
11.6.1 Intravenous
11.6.2 ICV
11.6.3 Intracerebral
11.6.4 Intracisternal
11.7 Basis Point Share (BPS) Analysis by Type
11.8 Absolute $ Opportunity Assessment by Type
11.9 Market Attractiveness Analysis by Type
11.10 Asia Pacific Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Applications
11.10.1 Mucopolysaccharidosis I
11.10.2 Mucopolysaccharidosis II
11.10.3 Mucopolysaccharidosis III A
11.10.4 Mucopolysaccharidosis III B
11.11 Basis Point Share (BPS) Analysis by Applications
11.12 Absolute $ Opportunity Assessment by Applications
11.13 Market Attractiveness Analysis by Applications
Chapter 12 Latin America Gene Therapy for Mucopolysaccharidosis Analysis and Forecast
12.1 Introduction
12.2 Latin America Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Country
12.2.1 Brazil
12.2.2 Mexico
12.2.3 Rest of Latin America (LATAM)
12.3 Basis Point Share (BPS) Analysis by Country
12.4 Absolute $ Opportunity Assessment by Country
12.5 Market Attractiveness Analysis by Country
12.6 Latin America Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Type
12.6.1 Intravenous
12.6.2 ICV
12.6.3 Intracerebral
12.6.4 Intracisternal
12.7 Basis Point Share (BPS) Analysis by Type
12.8 Absolute $ Opportunity Assessment by Type
12.9 Market Attractiveness Analysis by Type
12.10 Latin America Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Applications
12.10.1 Mucopolysaccharidosis I
12.10.2 Mucopolysaccharidosis II
12.10.3 Mucopolysaccharidosis III A
12.10.4 Mucopolysaccharidosis III B
12.11 Basis Point Share (BPS) Analysis by Applications
12.12 Absolute $ Opportunity Assessment by Applications
12.13 Market Attractiveness Analysis by Applications
Chapter 13 Middle East & Africa (MEA) Gene Therapy for Mucopolysaccharidosis Analysis and Forecast
13.1 Introduction
13.2 Middle East & Africa (MEA) Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Country
13.2.1 Saudi Arabia
13.2.2 South Africa
13.2.3 UAE
13.2.4 Rest of Middle East & Africa (MEA)
13.3 Basis Point Share (BPS) Analysis by Country
13.4 Absolute $ Opportunity Assessment by Country
13.5 Market Attractiveness Analysis by Country
13.6 Middle East & Africa (MEA) Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Type
13.6.1 Intravenous
13.6.2 ICV
13.6.3 Intracerebral
13.6.4 Intracisternal
13.7 Basis Point Share (BPS) Analysis by Type
13.8 Absolute $ Opportunity Assessment by Type
13.9 Market Attractiveness Analysis by Type
13.10 Middle East & Africa (MEA) Gene Therapy for Mucopolysaccharidosis Market Size Forecast by Applications
13.10.1 Mucopolysaccharidosis I
13.10.2 Mucopolysaccharidosis II
13.10.3 Mucopolysaccharidosis III A
13.10.4 Mucopolysaccharidosis III B
13.11 Basis Point Share (BPS) Analysis by Applications
13.12 Absolute $ Opportunity Assessment by Applications
13.13 Market Attractiveness Analysis by Applications
Chapter 14 Competition Landscape
14.1 Gene Therapy for Mucopolysaccharidosis Market: Competitive Dashboard
14.2 Global Gene Therapy for Mucopolysaccharidosis Market: Market Share Analysis, 2019
14.3 Company Profiles (Details – Overview, Financials, Developments, Strategy)
14.3.1 Sangamo Therapeutics
14.3.2 Swedish Orphan Biovitrum
14.3.3 uniQure
