Global Gene Therapy Market (2023-2028) by Type, Vector, Therapeutic Area, Delivery Mode, Route of Administration, and Regional Analysis, and AI and Sustainability, and Market Share Analysis

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Publish Date

04-03-2024

Report ID

JI-H-005

Pages

180

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Introduction

The Global Gene Therapy Market is set to surge from USD xx Bn in 2023 to a projected USD xx Bn by 2028, growing at a CAGR of xx%.

Advancements in gene editing technologies, such as CRISPR-Cas9, TALENs, and zinc finger nucleases, have revolutionized the field of gene therapy. These technologies allow for precise modification of genetic material, improving the efficacy and safety of gene therapies. The continuous refinement and development of these tools contribute to the expansion of treatment options and the potential for addressing a broader range of genetic disorders.

The integration of cutting-edge technologies enhances the precision and efficiency of gene therapy interventions, fostering innovation and expanding the scope of treatable conditions. This technological progress is a crucial driver in making gene therapies more accessible and effective.

The overall growth in technology adoption across various sectors, including healthcare, is fueling the market. Improvements in bioprocessing, manufacturing techniques, and delivery systems are streamlining production processes and making gene therapies more scalable and cost-effective. Additionally, advancements in diagnostic tools contribute to better patient selection and personalized treatment strategies.

The synergy between gene therapy and broader technological advancements creates a conducive environment for market growth. Enhanced manufacturing capabilities and personalized treatment approaches contribute to the market’s robust expansion.

There is an increasing demand for innovative therapeutic approaches, especially in the treatment of genetic disorders and certain types of cancers. Cell and gene therapies offer a promising solution by addressing the root cause of diseases at the genetic level. The rising awareness among healthcare professionals and patients about the potential benefits of these therapies creates a favorable market environment.

The escalating demand reflects a paradigm shift in the treatment landscape, presenting a substantial opportunity for market players. Capitalizing on this demand necessitates strategic investments in research, development, and production capabilities.

 

Segment Analysis for Gene Therapy Market

The Global Gene Therapy Market is segmented based on Type, Vector, Therapeutic Area, Delivery Mode, Route of Administration, and Geography.

The Global Gene Therapy Market, By Vector, is segmented based on Viral Vectors and Non-Viral Vectors.

The market often utilizes vectors to deliver genetic material into target cells. Researchers widely use viral vectors due to their efficient capability to transport genetic material into cells.. The viral vector segment consists of two primary types: Retroviral Vectors and Adeno-Associated Viral Vectors. Retroviral vectors are a class of viral vectors that can integrate their genetic material into the host cell’s genome. This integration is a key feature for long-term gene expression. Retroviral vectors are further segmented into Gamma Retroviral Vectors and Lentiviral Vectors.

Gamma Retroviral Vectors: Gamma retroviral vectors, belonging to the Retroviridae family, possess the capability to integrate into the genome of the host cell. These vectors find application in gene therapy, specifically in ex vivo gene therapy, where cells undergo modification outside the body before reintroduction.

Lentiviral Vectors: Lentiviral vectors, a subtype of retroviral vectors, are known for their ability to infect both dividing and non-dividing cells. This makes them suitable for a broader range of therapeutic applications, including in vivo gene therapy. Lentiviral vectors have gained popularity for their potential in treating genetic disorders, immunodeficiencies, and certain cancers.

Retroviral vectors, including both Gamma Retroviral Vectors and Lentiviral Vectors, provide a stable and long-term expression of the therapeutic gene, making them suitable for addressing chronic conditions. Lentiviral vectors, with their ability to infect non-dividing cells, offer versatility in therapeutic applications, expanding the range of treatable diseases. Safety concerns related to the potential for random integration into the host genome are associated with retroviral vectors. Ensuring targeted and controlled integration remains a key challenge for these vectors. Lentiviral vectors, while versatile, require careful consideration of their potential immunogenicity and off-target effects. Ongoing research and development efforts focus on improving the safety and efficiency of retroviral vectors, addressing concerns related to potential adverse effects. The versatility of lentiviral vectors opens up opportunities for the treatment of a diverse array of genetic disorders, providing a platform for continued innovation in gene therapy.

 

Regional Analysis for Gene Therapy Market

The Americas, including North and South America, have been a significant hub for gene therapy research, development, and commercialization. The Americas, particularly the United States, has been at the forefront of conducting clinical trials. Regulatory bodies such as the U.S. Food and Drug Administration (FDA) have played a crucial role in facilitating the development and approval of gene therapies.

Notable gene therapies, such as Luxturna for a rare form of inherited blindness and Zolgensma for spinal muscular atrophy, have received FDA approval, indicating a positive trend in the market. The region has seen substantial investments from both public and private sectors in research and development. Biotechnology and pharmaceutical companies in the United States, in particular, actively involve in advancing gene therapies.

Advancements in gene editing technologies, such as CRISPR-Cas9, TALENs, and zinc finger nucleases, have fueled research and development efforts. These technologies offer precision and efficiency in modifying genetic material, expanding the scope of potential therapeutic applications. Gene therapy has moved beyond rare diseases to address a broader range of medical conditions. Ongoing research explores applications in oncology, cardiovascular diseases, neurodegenerative disorders, and infectious diseases.

 

List of Companies

The report provides profiles of the key companies, outlining their history, business segments, product overview, and company financials. Some companies from competitive analysis are Alnylam Pharmaceuticals, Inc., Amgen, Inc., Anges, Inc., Biogen, Inc., Biomarin Pharmaceutical Inc., etc.

 

Key Developments

EMA has recommended the approval of the first medicine using CRISPR/Cas9, a novel gene-editing technology. Health regulatory authorities have granted approval for Casgevy to treat patients aged 12 and older experiencing transfusion-dependent beta-thalassemia and severe sickle cell disease. This approval applies in situations where healthcare professionals consider hematopoietic stem cell transplantation appropriate, and a suitable donor is not available. This new therapy may free patients from the burden of frequent transfusions and painful vaso-occlusive crises that occur when sickled red blood cells block small blood vessels, and have the potential to significantly improve their quality of life.   December 2023

Sarepta Therapeutics in its fourth-quarter and full-year 2023 earnings report on Wednesday touted the strong market demand for its Duchenne muscular dystrophy gene therapy Elevidys, which has brought in just over USD 200 Mn since its launch in mid-2023.

In June 2023, Elevidys achieved the historic milestone of becoming the first-ever FDA-approved gene therapy for Duchenne muscular dystrophy (DMD), clearing the regulatory gauntlet. The treatment works by delivering a functional copy of the dystrophin gene into the patients’ muscle tissues, which addresses the genetic root of DMD. Mutations to this gene lead to the muscle weakness and developmental delay associated with the disease. – February 2024

 

Frequently Asked Questions

How Much Is The Global Gene Therapy Size?

The Global Gene Therapy is set to surge from USD xx Bn in 2023 to a projected USD xx Bn by 2028, growing at a CAGR of xx%.

What Is The Major Global Gene Therapy Driver?

  • Increasing Investments in Gene Therapy Research

  • Growing Technological Advancements

What are the Major Gene Therapy Opportunities?

  • CRISPR Gene Editing-based Therapeutics has Long-term Growth Opportunity

Who Are The Gene Therapy Players?

Alnylam Pharmaceuticals, Inc., Amgen, Inc., Anges, Inc., Biogen, Inc., Biomarin Pharmaceutical Inc., Bluebird Bio, Inc., Bristol-Myers Squibb, etc.

1. Introduction
1.1 Scope of the Study
1.2 Purpose of the Study
1.3 Limitations of the Study
1.4 Currency

2. Executive Summary
2.1 Market Definition
2.2 Market Size and Segmentation
2.3 Insights for CXOs

3. Market Forces
3.1 Drivers
3.1.1 Increasing Regulatory Approvals for Gene Therapy Products
3.1.2 Increasing Investments in Gene Therapy Research
3.1.3 Growing Technological Advancements
3.2 Restraints
3.2.1 High Costs of Gene Therapy Products
3.3 Opportunities
3.3.1 CRISPR Gene Editing-based Therapeutics has Long-term Growth Opportunity
3.3.2 Rising Demand for Cell and Gene Therapies
3.3.3 Increasing Focus on Precision and Medicine
3.4 Challenges
3.4.1 COGS Remain Partially High Due to Low and Variable Yields
3.4.2 Supply Chain Challenges

4. AI and Sustainability Footprints
4.1 Market Trends of AI and Sustainability
4.2 Application of AI - AI Adoption Levels
4.3 Environmental and Emission Reduction Initiatives

5. Market Analysis
5.1 Government Regulations
5.2 Value Chain Analysis
5.3 PESTLE Analysis
5.4 Porter's Five Forces Analysis
5.5 Technology Trends
5.6 Ansoff Analysis

6. Market Segmentations
6.1 Global Gene Therapy Market, By Type
6.1.1 Market Overview, Size and Forecast
6.1.2 Gene Silencing
6.1.3 Cell Replacement
6.1.4 Gene Augmentation
6.1.5 Other Therapies

6.2 Global Gene Therapy Market, By Vector
6.2.1 Market Overview, Size and Forecast
6.2.2 Viral Vector
6.2.2.1 Retroviral Vectors
6.2.2.1.1 Gamma Retroviral Vectors
6.2.2.1.2 Lentiviral Vectors
6.2.2.2 Adeno-Associated Viral Vectors
6.2.2.3 Other Viral Vectors
6.2.3 Non-Viral Vectors
6.2.3.1 Oligonucleotides
6.2.3.2 Other Non-Viral Vectors

6.3 Global Gene Therapy Market, By Therapeutic Area
6.3.1 Market Overview, Size and Forecast
6.3.2 Neurology
6.3.3 Oncology
6.3.4 Heptalogy
6.3.5 Other Therapeutic Areas

6.4 Global Gene Therapy Market, By Delivery Method
6.4.1 Market Overview, Size and Forecast
6.4.2 In Vivo
6.4.3 Ex Vivo

6.5 Global Gene Therapy Market, By Route of Administration
6.5.1 Market Overview, Size and Forecast
6.5.2 Intravenous
6.5.3 Other Routes of Administration

7. Region Analysis of the Global Gene Therapy Market
7.1 Americas
7.1.1 Brazil
7.1.2 Canada
7.1.3 Mexico
7.1.4 US
7.1.5 Others
7.2 Europe
7.2.1 France
7.2.2 Germany
7.2.3 Italy
7.2.4 Russia
7.2.5 United Kingdom
7.2.6 Others
7.3 Middle East & Africa
7.3.1 Israel
7.3.2 Nigeria
7.3.3 Saudi Arabia
7.3.4 South Africa
7.3.5 UAE
7.3.6 Others
7.4 Asia
7.4.1 Australia – New Zealand
7.4.2 China
7.4.3 India
7.4.4 Japan
7.4.5 South Korea
7.4.6 Others

8. Competitive Analysis
8.1 Market Share Analysis
8.2 Alnylam Pharmaceuticals, Inc.
8.3 Amgen, Inc.
8.4 Anges, Inc.
8.5 Biogen, Inc.
8.6 Biomarin Pharmaceutical Inc.
8.7 Bluebird Bio, Inc.
8.8 Bristol-Myers Squibb
8.9 F. Hoffmann-La Roche AG
8.10 Ferring B.V.
8.11 Gilead Sciences, Inc.
8.12 Jazz Pharmaceuticals PLC
8.13 Johnson & Johnson
8.14 Krystal Biotech, Inc.
8.15 Meiragtx Holdings PLC
8.16 Novartis
8.17 Orchard Therapeutics, PLC
8.18 Pfizer, Inc.
8.19 Regenxbio
8.20 Sangamo Therapeutics, Inc.
8.21 Sarepta Therapeutics, Inc.
8.22 Shanghai Sunway Biotech Co. Ltd.
8.23 Sibiono Genetech Co. Ltd.
8.24 Ultragenyx Pharmaceutical, Inc.
8.25 Uniqure N.V.
8.26 Vertex Pharmaceuticals, Inc.

9. Case Studies

10. Appendix
10.1 Research Methodology
10.2 Assumptions for the Report
10.3 List of Abbreviations

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