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Monogenetic Disease Therapy Market Report


Monogenetic Disease Therapy Market, by Inheritance Pattern (Chromosomal and Autosomal), by Therapy Type (Surgical, EBRT, Blood Transfusion, Pharmacological, Gene Therapies), by Application (Phenylketonuria, SCID, Cystic Fibrosis, Sickle Cell Anemia), Regional Analysis (North America, Europe, Asia-Pacific, LAMEA) – Global Opportunity Analysis and Industry Forecast, 2019–2026


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Feb 2020

Global Monogenetic Disease Therapy Market Overview 2026:

Monogenetic diseases are the outcome of the modification in the DNA sequence of a gene. A changed gene hampers the traditional working of the protein which is present in the arrangement which in turn results in several kinds of monogenetic disorders, for instance, sickle cell disease, cystic fibrosis, SCID, and Gaucher disease among others. The monogenetic disease therapy market is anticipated to upsurge during the projected period; this is majorly due to the growth in the occurrence of monogenetic diseases, along with advanced treatment technologies.

Driving Factors: Growing research and development activities are anticipated to drive the Global Monogenetic Disease Therapy Market.

The monogenetic disease therapy market is projected to boost in recent years, owing to the increasing incidence of monogenetic diseases across the globe. As per the article published by the World Health Organization (WHO), the total incidence of monogenetic diseases during the time of birth is about 10/1000. Growing clinical trials and R&D activities along with its therapeutic treatments are projected to impel the monogenetic diseases therapy market. In addition, increasing awareness about genetic disease and the presence of major market players globally such as Pfizer Inc., Bayer AG, and Sanofi among others those are investing hugely in developing advanced therapies, vaccines, and innovative bio-instruments to treat the people suffering from monogenetic diseases are expected to drive the market growth.

High treatment cost is anticipated to restrain the market growth in the near future

Moreover, high cost associated with the monogenetic disease therapy coupled with lesser success rates of treatments such as gene therapy is expected to hinder the development of the global monogenetic disease therapy market.

Chromosomal disease therapy segment is predicted to be most lucrative in the estimated timeframe

The monogenetic disease therapy market-based in inheritance pattern is bifurcated into Chromosomal and Autosomal.  The autosomal monogenetic disease therapy segment is projected to hold the largest market share in 2018. The growth is majorly attributed to the increase in the number of people suffering from autosomal monogenetic diseases such as Cystic Fibrosis Sickle cell anemia and Tay Sachs disease. Chromosomal disease therapy is projected to increase during the forecasted timeframe, due to the increasing incidence of hunter disease, hemophilia, and others. 

Gene therapy segment is anticipated to dominate the therapy type segment in the near future

Pharmacological and surgical therapies that are used for curing monogenetic diseases are anticipated to lead the global monogenetic disease therapy market, owing to the growing preference of this type of therapy among the patients. In February 2018, U.S. FDA permitted a mixture of tezacaftor and ivacaftor manufactured by Vertex Pharmaceutical, to cure cystic fibrosis. Gene therapy segment is estimated to observe a swift growth during 2019-2026; this growth is majorly attributed to its effectiveness in treating people suffering from monogenetic diseases such as SCID and CF. For example, Glybera is the foremost clinically permitted gene therapy in the European region that uses an adeno-associated virus (AAV) vector drug to fight the lipoprotein lipase deficiency.

Cystic fibrosis segment accounted for the highest Monogenetic Disease Therapy Market share by 2026

The monogenetic disorder therapy market on the basis of the application was divided into Phenylketonuria, SCID, Cystic Fibrosis, and Sickle Cell Anemia. Cystic fibrosis will account for the major revenue share; the growth is due to the higher prevalence of monogenetic diseases worldwide, along with growing research and development activities for the treatment of cystic fibrosis. As per the article published by Cystic Fibrosis Foundation (CFF), approximately more than 70,000 people globally are living with cystic fibrosis. Growth in the occurrence of monogenic diseases such as Thalassaemia, specifically in developing nations such as India, Iran, and Pakistan, is also projected to upsurge the monogenetic disease therapy market during the forecast period.

Regional Insights:

North America's monogenetic disease therapy market is expected to lead the global monogenetic disease therapy market, owing to the existence of a number of key market players such as Amgen and Abbott Laboratories in this region. Rising clinical trials in North America region is additionally one of the key growth factors for North America to dominate the global monogenetic disease therapy market. As per NCBI, 66.81% of total gene therapies took place in the U.S., between 1989 to 2015.

Monogenetic disease therapy market in Europe and the Asia Pacific regions are projected to increase during the projected period, due to the rising prevalence of monogenetic diseases, along with growing people’s healthcare expenditure. To explore more about the Monogenetic Disease Therapy Market, Connect with Analyst

Key players in the Global Monogenetic Disease Therapy Market:

Product development and joint ventures are the most common strategies followed by the market players

Key players in the Global Monogenetic Disease Therapy Market
Source: Research Dive Analysis

Major players in the global monogenetic disease therapy market include Bayer AG, UniQure, Bristol-Myers Squibb, BAG Healthcare Gmbh, Abbott Laboratories, Novartis, Vertex Pharmaceuticals, Pfizer Inc., F. Hoffmann-La Roche Limited, Bluebird Bio, Grifols S.A, and Sanofi S.A. These key players are majorly focusing on collaborations, partnerships, mergers and acquisitions and other activities to strengthen their market presence. For example, in August 2018, Mustang Bio, a U.S. headquartered pharmaceutical organization signed a license agreement with St. Jude Children’s Research Hospital to improve the ex-vivo lentiviral gene therapy for X-linked SCID treatment. With the help of this merger, the Mustang Bio has spread its product portfolio into gene therapy for patients suffering from X-SCID.



  Historical Market Estimations


  Base Year for Market Estimation


  Forecast timeline for Market Projection


  Geographical Scope

  North America, Europe, Asia-Pacific, LAMEA

  Segmentation by Inheritance Pattern

  • Chromosomal

  • Autosomal

  Segmentation by Therapy Type

  • Surgical

  • ERBT

  • Blood Transfusion

  • Pharmacological

  • Gene Therapies

  Segmentation by Application

  • Phenylketonuria

  • SCID

  • Cystic Fibrosis

  • Sickle Cell Anemia

  Key Countries Covered

U.S., Canada, Germany, France, Spain, Russia, Japan, China, India, South Korea, Australia, Brazil, and Saudi Arabia

  Key Companies Profiled

  • Bayer AG

  • UniQure

  • Bristol-Mayers Squibb

  • BAG Healthcare GmbH

  • Abbott Laboratories

  • Novartis Pharmaceuticals

  • Pfizer Inc.

  • F. Hoffmann-La Roche Limited

  • Bluebird Bio

  • Grifols S.A.

  • Sanofi S.A.

  • Mustang Bio

Source: Research Dive Analysis


Frequently Asked Questions

A. Bayer Ag, BAG Healthcare GmbH, and Vertex Pharmaceuticals are some of the key players in the global expanded monogenetic disease therapy market.

A. Asia-Pacific possesses great investment opportunities for investors to witness the most promising growth in the coming years.

A. Product development and joint ventures are the key strategies opted by the operating companies in this market.

A. Vertex Pharmaceuticals, Bayer AG, UniQure are investing more in R&D activities for developing new products and technologies.

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