Gene Therapy for Inherited Genetic Disorders Market - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast 2018 - 2028

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Gene Therapy for Inherited Genetic Disorders Market - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast 2018 - 2028

Status : August, 2020 | Medical Devices | PPT format

Report Digest


Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer. 

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.  

Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease). 

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

 Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practical—and accessible—gene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations. 

Some of the most prominent competitors operating in the competitive landscape of global gene therapy for inherited genetic disorders market include –

  • Spark Therapeutics Inc.
  • Orchard Therapeutics
  • Novartis AG
  • Bluebird bio Inc.
  • BioMarin Pharmaceutical

Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market. 

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful. 

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

The study presents a granular assessment and quantitative evaluation of:

  • Key growth factors and prominent trends
  • Size and share of key segments in overall market
  • Key challenges and winning imperatives
  • Factors shaping competitive landscape
  • Insights into how contours of market will change in coming years
  • Technological advances
  • New avenues and imminent investment pockets
  • Regulatory landscape shaping current and future strategies of companies

The region-wise assessment includes market dynamics in:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The study is prepared with the help of an extensive primary and secondary research. Primary research includes but not limited to interactions, interviews, surveys, and events. Opinions of policy makers and industry experts are carefully integrated into the insights to complement primary research. Volumes of secondary data gleaned through renowned paid sources, annual statements, reputed journals, and government documents offer insights into current dynamics and key evolution trajectories the market is likely to assume. The research employs various industry-wide analytics methods and well-established methodologies to ascertain the attractiveness of key segments. Further, it evaluates the lucrativeness of key regions and factors underlying their maturity. The insights notably include a detailed analysis of forces behind vendor differentiation and offers insight into investment-decision making by prominent players.

The report strives to enlighten businesses and industry stakeholders on multiple facets of the growth dynamic of the market, the key of which include answers to:

  • Which key factors will shape the competitive landscape?
  • Which trends will stimulate research and development activities in different geographies?
  • Which are the lucrative growth areas and what is the size of potential they are likely contribute to the overall market?
  • Which disruptions in market growth are likely to come with the new business models?
  • Which are some of the recent overhauls in policies that may change the course of the market significantly?
  • Which are the geographies that will form sizable sources of new streams to new and established players alike?
  • How are top players likely to realign their strategy to counter the forces of competition?
  • Which key strategies emerging players are likely to focus on to consolidate their position?
  • Which changes in end-user preferences might affect the status quo held by the leading players?
  • Which are some of the game-changing innovations influencing the projected share and revenue of the market and its segments?

Note: Utmost care has invariably been taken to present data and arrive at statistics at all the TMR Research reports. Meanwhile, some recent developments and new industry trends may take time to be included in the reports.