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Global Monogenetic Disease Therapy Market Size, Share, Industry Statistics Report, By Inheritance Pattern (Autosomal and Chromosomal); By Therapy Type (Pharmacological, Surgical, Blood Transfusion, EBRT, Gene Therapies, Others); By Application (Cystic Fibrosis, SCID, Sickle Cell Anemia, Phenylketonuria, Others) Regional Analysis (North America - U.S, Canada, Other) (Asia-Pacific - India, Japan, China, South Korea, Japan, Australia, and Other) (Europe - U.K., Germany, Netherlands, Lithuania, Austria, Belgium, Poland, Sweden, Spain, France and Other) (Middle East and Africa and RoW) - Global Industry Insights, Trends and Forecast, 2017-2025

Report Format : PDF
Published Date : Sep-2018
Pages : 200
Report ID : VSR00552

Global Monogenetic Disease Therapy Market Size showed crucial growth in the recent years and is anticipated to witness a steep rise by generating an estimated revenue of US$ xx.xx billion over the forecast period. According to latest study conducted by our analysts and industry experts, the global market is expected to grow at a CAGR of 7.6% during the forecast period that is 2017-2025. Monogenetic diseases result from the mutation or change in the DNA sequence of one gene. A mutated gene hinders the normal functioning of the protein present in its composition and hence results in various types of monogenetic disorders such as cystic fibrosis, sickle cell disease, Gaucher disease, SCID and others. The monogenetic disease therapy market is expected to grow during the forecast period, due to a rise in the incidence of monogenetic diseases, coupled with advancement in treatment technologies.

Global Market Growth Opportunities (Revenue, Growth) By 2017-2025

Monogenetic Disease Therapy Market Size

This global market study report analysis offers in-depth insights, revenue details, and other vital information regarding the global monogenetic disease therapy market, and the various trends, drivers, restraints, opportunities, and threats in the target market till 2025. The report offers insightful and detailed information regarding the various key players operating in the market, their financials, supply chain trends, technological innovations, key developments, apart from future strategies, acquisitions & mergers, and market footprint. The global monogenetic disease therapy market report has been segmented on the basis of type, application, and region. It includes the estimation of the market size, in terms of value, with respect to 4 main geographies, namely, North America, Europe, APAC, and RoW.   

Market Dynamics

The monogenetic disease therapy market is expected to grow during the forecast period, due to the rising prevalence of monogenetic diseases globally. According to the World Health Organization (WHO), the global prevalence of all monogenetic diseases at birth is approximately 10/1000. Increased research activities and clinical trials on monogenic diseases and its therapeutic treatments are further anticipated to drive the monogenetic diseases therapy market. According to NCBI, monogenetic diseases were the second most popular indication of gene therapies after cancer, accounted for 10% of the total gene therapy trials conducted between 1989 and 2015. Other driving factors for the growth of monogenetic disease therapy market include rising awareness towards genetic diseases, coupled with the presence of key healthcare market players such as Sanofi, Bayer AG, Pfizer Inc., and others who are determined to develop innovative therapies, advanced bio-instruments, and vaccines to treat monogenetic diseases. However, high therapy cost and lower commercial success rates of treatments like gene therapy can restrain the growth of global monogenetic disease therapy market.

By Inheritance Pattern Emphasis: On the basis of inheritance pattern, autosomal monogenetic diseases therapy market is expected to grow at a faster pace, due to rise in a number of autosomal monogenetic diseases like Cystic fibrosis (a most common type of monogenetic disease), Tay Sachs disease and Sickle cell anemia. According to the European Lung White Book, one in 25 people of European descent carries one mutant allele of CFTR, and one person among 2000-3000 people is affected by cystic fibrosis. Chromosomal disease therapy is also expected to grow during the forecast period, owing to the rising incidence of hemophilia, hunter disease, and others. Haemophilia is estimated to be afflicted in 1 in 10,000 people globally.

By Therapy Type Insights: Pharmacological and surgical therapies for treating monogenetic diseases are predicted to dominate the global market, due to their higher preference among patients. For instance, in February 2018, the U.S. FDA approved a combination of ivacaftor and tezacaftor, developed by Vertex Pharmaceutical, to treat cystic fibrosis. Gene therapy is another therapy segment which is expected to witness a rapid growth over the forecast period, owing to its efficiency in the treatment of monogenetic diseases like CF and SCID. For instance, Glybera, which is the first clinically approved gene therapy in the European Union, uses an adeno-associated virus (AAV) vector drug for correction of lipoprotein lipase deficiency and to correct cystic fibrosis, it uses repeated nebulization of liposomes encoding the cystic fibrosis transmembrane conductance regulator.

By Application Analysis: Monogenetic disorder therapy market for Cystic fibrosis (an autosomal recessive disease) will account for the major revenue share, due to a higher prevalence of the disease worldwide, coupled with increased research and development activities in the treatment of cystic fibrosis. According to the Cystic Fibrosis Foundation, more than 70,000 people worldwide are living with cystic fibrosis. Increase in a number of monogenic diseases like Thalassaemia, especially in developing countries like Pakistan and Iran, is also expected to boost the monogenetic disease therapy market. For instance – estimates in Iran, suggest that about 8,000 pregnancies remain at risk of getting alpha or beta thalassaemias annually.

Regional Analysis

The global monogenetic disease therapy market is divided into regions that are North America - U.S, Canada, Other; Asia-Pacific -India, Japan, China, Australia, Other; Europe - UK, Russia, France, Brazil, Other; Middle East and Africa. Every region analysis details in sense of growth, revenue, volume, challenges, and opportunities with the regional and global key players. North America is expected to dominate the global monogenetic disease therapy market, due to the presence of a number of key players like Abbott Laboratories and Amgen in the region. Increased clinical trials in this region, is also one of the major reasons for the dominance of North America in the global monogenetic disease therapy market. According to NCBI, 66.81% of total gene therapies were conducted in the U.S., from 1989 to 2015.

Global Monogenetic Disease Therapy Market Size, Share Analysis By Region 2017-2025

Monogenetic Disease Therapy Market Share

Monogenetic disease therapy market in Europe and the Asia Pacific is expected to grow during the forecast period, owing to the increased prevalence of monogenetic diseases, coupled with rising people’s healthcare expenditure. According to the Institute for Health Metrics and Evaluation (IHME), lower-middle income countries and low-income countries are expected to spend US$ 507.0 and US$ 164.0 per person on health respectively, in 2040. Middle East and Africa held the measurable growth in the global market in 2018 and will expand at the significant CAGR during the forecast years.    

Global Monogenetic Disease Therapy Market Study Objective

1. To forecast the market size of monogenetic disease therapy market, along with describing and defining the market, in terms of volume and revenue as well as market opportunities for stakeholders, in order to provide a competitive landscape for global market players.

2. Market analysis and forecast of monogenetic disease therapy market on the basis of region, substrate and application or end-use industry. To profile major market players, and to analyze their market shares and essential competencies comprehensively.

3. To analyze and elaborate significant factors that will have major impact on the growth of monogenetic disease therapy market, such as drivers, restraints, challenges and opportunities.

4. Regional analysis of monogenetic disease therapy market, to induce market size of five global major regions, which include, Europe, North America, South America, Middle East & Africa and Asia Pacific.

5. Strategic analysis of micromarkets, in terms of individual prospects, opportunities, growth trends and their involvement in overall market.

6. To provide detailed analysis of competitive and strategic developments in the monogenetic disease therapy market like new product launch, partnership & agreement, investment & expansion, and company merger & acquisitions.

Key Players Landscape

Key market players operating in global monogenetic disease therapy market include Abbott Laboratories, BAG Healthcare Gmbh, Novartis, Pfizer Inc., Vertex Pharmaceuticals, Bristol-Myers Squibb, F. Hoffmann-La Roche Limited, Bayer AG, Grifols S.A, Bluebird Bio, UniQure, and Sanofi S.A. Market players, clinical research organizations and hospitals are focusing on collaborations to develop and commercialize therapies for the treatment of monogenetic diseases. For instance, in August 2018, Mustang Bio, a U.S. based pharmaceutical firm entered into an exclusive license agreement with St. Jude Children’s Research Hospital to develop the ex-vivo lentiviral gene therapy for X-linked SCID (commonly known as Bubble Boy Disease) treatment. Through this merger, Mustang Bio aims to expand their pipeline into gene therapy for patients with X-SCID.

Global Monogenetic Disease Therapy Market Study Report Offer You!

The global market study report gives an idea about market size, share, stock, equity, investment and total price analysis. Also, the report provides comprehensively industry insights with all major factors such as market credit, allocation, demand, distribution, market scope, key players, end user & applications, provisions, livestock, commodities and industry economics analysis, future growth & forecast. The global market report includes all depth data like pre-market emphasis, market survey, market investment, industry advice, portfolio, finance, access, trade, market exploration, industry review and market predication & projection to develop effective strategies for optimizing their market position.

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Global Monogenetic Disease Therapy Market Size, Share, Industry Statistics Report, By Inheritance Pattern (Autosomal and Chromosomal); By Therapy Type (Pharmacological, Surgical, Blood Transfusion, EBRT, Gene Therapies, Others); By Application (Cystic Fibrosis, SCID, Sickle Cell Anemia, Phenylketonuria, Others) Regional Analysis (North America - U.S, Canada, Other) (Asia-Pacific - India, Japan, China, South Korea, Japan, Australia, and Other) (Europe - U.K., Germany, Netherlands, Lithuania, Austria, Belgium, Poland, Sweden, Spain, France and Other) (Middle East and Africa and RoW) - Global Industry Insights, Trends and Forecast, 2017-2025

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Global Monogenetic Disease Therapy Market Size, Share, Industry Statistics Report, By Inheritance Pattern (Autosomal and Chromosomal); By Therapy Type (Pharmacological, Surgical, Blood Transfusion, EBRT, Gene Therapies, Others); By Application (Cystic Fibrosis, SCID, Sickle Cell Anemia, Phenylketonuria, Others) Regional Analysis (North America - U.S, Canada, Other) (Asia-Pacific - India, Japan, China, South Korea, Japan, Australia, and Other) (Europe - U.K., Germany, Netherlands, Lithuania, Austria, Belgium, Poland, Sweden, Spain, France and Other) (Middle East and Africa and RoW) - Global Industry Insights, Trends and Forecast, 2017-2025

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