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 : May, 2021 | Medical Devices

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.

This study provides a particularized anatomy according to the L.E.A.P mechanism

  • Latest trends and cardinal growth prospects
  • Existing and future insights in growth
  • Ascensions in technology
  • Pain points

The regional analysis offers market assays across:

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

The study, prepared through the L.E.A.P mechanism adds a dimension of infallibility and assures precise information on all the growth dynamics.

Latest Trends and Cardinal Growth Prospects

The study assists in burnishing the knowledge of the stakeholder in terms of the emerging trends. The study provides an expansive coverage on the latest developments and novel profit-yielding sources that augur well with the market. A dedicated and determined team of experts conduct an out-and-out research on diverse aspects to offer ultimate breakthroughs in the market.

Existing and Future Insights in Growth

The interfusion of ubiquitous primary and secondary research through modern tools invites perfection and accuracy in every aspect. Primary research is circumspectly conducted through exhaustive interactions and interviews of experts. These valuable opinions are then neatly inducted in the report to allow the stakeholder for reaping the benefits. A treasure of secondary data is extracted via famed paid sources, yearly estimates and statements, renowned journals, and government documents. These sources assist in creating a robust database comprising existing and subsequent growth dynamics.

Ascensions in Technology

Diverse technological advancements and cutting-edge innovations in the market have been covered in this study to assist the stakeholder in research and development activities. Recent upgrades have also been added in the study, thus helping the stakeholder to paint the strokes of growth on the canvas of the market.

Pain Points

The study presents a plethora of challenges and obstacles that can hinder the growth of the market to allow the stakeholder to prepare their strategies accordingly.

The world ushered in the new decade with the shadow of the COVID-19 pandemic. The outbreak has damaged the growth prospects of several businesses and has crippled the entire economy. The study is in tandem with the changing dynamics of the market due to the SARS-CoV-2 outbreak. The study focuses on the coronavirus pandemic and analyses every aspect that impacts the growth positively or negatively.

An aerial perspective of the regions covered in this study enables the stakeholder to design strategies accordingly. The 360-degree assessment of every aspect associated with growth offers information on a rainbow of growth opportunities available across the market.

The exhaustive study motivates the stakeholder to take the 'LEAP' of faith and achieve success!

This report is the key to questions such as:

What are the prominent factors that will help in reshaping the market growth?

Which trends have the potential to bring expansive growth for the market?

What are the necessary strategies to overcome the ill-effects of the COVID-19 pandemic?

Which regions will emerge as champion growth contributors for the market?

Which aspects will serve as game-changers for the market?

What are the latest innovations in the market?

Which factors will hinder the growth of the market?