Global Gene Therapy Market Opportunity & Clinical Trials Insight, 2026 -

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Report Highlights

  • Global Gene Therapy Market Opportunity: > US$ 8 Billion
  • Global Gene Therapy Clinical Pipeline: > 1000 Therapies In Clinical Trials
  • Commercially Available Gene Therapies: 11
  • USA Dominates Global Gene Therapy Pipeline: > 500 Therapies In Clinical Trials
  • Cancer Accounts For 50% of Global Gene Therapy Trials

Global Gene Therapy Market Opportunity & Clinical Trials Insight, 2026 is poised towards the current market trends followed by the therapy since its advent at international level and delivering a summarized information about the whole concept of the gene therapy in order to restore the gene functions by manipulating the genetic code present within the genome.

The report discusses the current available gene therapy agents with their complete data regarding the dosage, sales and pricing compiled from multiple sources and from publications by companies that are thriving the market of gene therapy. The entire clinical and non-clinical framework along with the countries encompassed with the presence of agents is also discussed. The report highlights the need of such advanced technique and the entire huge breakthrough the technique is going through.

The whole arena of gene therapy came into limelight when the therapy treated the first patient having ADA-SCID. Since then, the fundamental benefits received from the therapy never got limited. The therapy has blessed the patients' many agents with unique strategies and nonetheless many efforts made by the researchers are underway to get utilized against the diseases that have no other viable treatment. The therapy can alter the target DNA sequence by silencing, replacing or by manipulating the level of the substitute protein in order to restore the patient's body against the target disease.

As there are various diseases that are associated with cancer, therefore the technology provides a potential treatment for the patient and it is expected that the therapy will provide deep insight into the genetic diseases and its treatment in future too. For the future, the gene therapy process is believed to be at a crucial position and will undoubtedly revolutionize the clinical, preclinical and technical techniques utilized in the laboratories. The editing of specific genes present in the pool of genome is considered as a groundbreaking discovery like the discovery of vaccines and antibiotics. It is just a matter of time that the technology will be categorized as a dominating one over the other available technologies.

Gene therapy has been designed such that it can provide relief to the patients with rare genetic disease with one approach i.e. manipulation at the genetic level. One approach of the technique gets investigated for a number of results which in-turn results in the approval of several gene therapy agents in a regulated way. In exchange, the therapy also entails for a cheap and one-time treatment- single injection or infusion when compared with the many years of expensive ongoing treatment.

The advent of the therapy for the well-being of the patients has definitely shifted the timings of the healthcare costs. The novel gene therapy agents anticipation in the market has successfully determined the value of the gene therapy and its urgency. The value-based research of the therapy has gained the interest of the clinicians and researchers ensuring the best of the benefits from the therapy. The comparative effectiveness and cost-effectiveness have inclined the dependency of the patients towards gene therapy while gaining excess commercial success in the global market.

Key Topics Covered

1. Introduction to Gene Therapy

1.1 Gene Therapy: An Overview

1.2 History of Development

2. Gene Therapy & its Impact on Genetic Alterations

2.1 Gene Therapy as a Mastermind of Genetic Revolution

2.2 Manipulations at Molecular Level by Gene Therapy

3. Working Mechanism of Gene Therapy

3.1 Classification of Gene Therapy on the Basis of Cell Type

3.1.1 Somatic Gene Therapy

3.1.2 Germline Gene Therapy

3.2 Classification on the Basis of Gene Delivery Mechanism

3.2.1 Ex-Vivo Delivery of the Gene

3.2.2 In-Vivo Delivery of the Gene

3.3 Classification on the Basis of Gene Delivery Methods

3.3.1 Viral Methods

3.3.2 Non-Viral Methods

3.4 Classification on the Basis of End-Results

3.4.1 Augmentation Gene Therapy

3.4.2 Targeted Gene Therapy

4. Molecular Tools Available for Gene Therapy

4.1 Targeting Specific Loci with ZFN's

4.2 Introduction of TALEN's as a Gene Therapy Tool

4.3 Development of CRISPR to Mediate Precise Genome Editing

5. Strategies Opted by Gene Therapy

5.1 Replacement of Defective Genes with Healthy Genes

5.2 Fixing Mutated Genes by Gene Therapy Molecular Tools

5.3 Gene Therapy Modifying the Diseased Cells & Making them Evident against Immune Cells

6. Gene Therapy & the Journey of Clinical Trials Associated

6.1 Trends Associated with Clinical Research & Trials

6.1.1 Clinical Trials Regarding Gene Therapy: Disease Analysis

6.1.2 Clinical Trials Regarding Gene Therapy: Vector Analysis

6.1.3 Clinical Studies Regarding Gene Therapy: Global Analysis

6.1.4 Clinical Trials Regarding Gene Therapy: Gene Type Analysis

6.1.5 Clinical Trials Regarding Gene Therapy: Current Status

6.1.6 Clinical Trials Regarding Gene Therapy: Gender Analysis

6.1.7 Clinical Trials Regarding Gene Therapy: Funding Analysis

7. Approach of Gene Therapy Against Cancer

7.1 Clinical Efficacy of Gene Therapy for Cancer Cells

7.2 Gendicine: A Wide Spectrum Anti-Cancer Gene Therapy Agent

7.3 Rexin-G for Osteosarcoma & Soft Tissue Sarcoma

7.4 Kymriah: A Breakthrough Gene Therapy Product for B-Cell Acute Lymphoblastic Leukemia

7.5 Yescarta: Gene Therapeutic Approach for B-Cell Lymphoma

7.6 Oncorine Approval by Shanghai Sunway Biotech Co. Ltd.

7.7 Gene Therapy Product Imlygic against Melanoma

8. Gene Therapy for the Treatment of Neuromuscular Diseases - Spinal Muscular Atrophy & Duchenne Muscular Dystrophy

8.1 Clinical Approach of Gene Therapy for Duchenne Muscular Dystrophy

8.2 Clinical Approach of Gene Therapy for Spinal Muscular Atrophy

8.3 Spinraza - First Approved Gene Therapy Product for Spinal Muscular Atrophy

8.4 Zolgensma Treatment for Spinal Muscular Atrophy Patients

8.5 Eteplirsen - A New Hope for the Patients with Duchenne Muscular Dystrophy

9. First Targeted Treatment for Rare Inherited Retinal Dystrophy

9.1 Clinical Efficacy of the Gene Therapy Products

9.2 Luxturna for the Treatment of RPE65 Mutation Associated Retinal Dystrophy

10. Gene Therapy Mediated Treatment of Peripheral Artery Disease

10.1 Clinical Interference for Cardiovascular Disorders Gene Therapy

10.2 Neovasculgen Gene Therapy in Cardiovascular Diseases

11. Correcting Severe Combined Immunodeficiency - Adenosine Deaminase Deficiency through Gene Therapy

11.1 Clinical Potential of Gene Therapy Therapeutic Approach

11.2 Strimvelis Benefits for Rare Disease: Adenosine Deaminase Deficiency- Severe Combined Immunodeficiency

12. Success of Gene Therapy over Other Diseases

13. Availability & Revolution of Gene Therapy Drugs in the Market

14. Gene Therapy Products Dosage & Price Analysis

14.1 Kymriah

14.2 Yescarta

14.3 Spinraza

14.4 Zolgensma

14.5 Luxturna

14.6 Strimvelis

14.7 Eteplirsen

14.8 Imlygic

15. Global Gene Therapy Clinical Trials Insight

15.1 By Phase

15.2 By Country

15.3 By Formulation

15.4 By Company

15.5 By Target

15.6 By Indication

16. Global Gene Therapy Clinical Trials By Company, Indication & Phase

16.1 Research

16.2 Preclinical

16.3 Clinical

16.4 Phase-0

16.5 Phase-I

16.6 Phase-I/II

16.7 Phase-II

16.8 Phase-II/III

16.9 Phase-III

16.10 Preregistration

16.11 Registered

17. Marketed Gene Therapy Clinical Insight


17.2 Kymriah


17.4 Gendicine


17.6 Glybera



17.9 Neovasculgen

17.10 Zalmoxis

17.11 Rexin-G

18. Global Market Landscape of Gene Therapy

18.1 Gene Therapy Market Overview

18.2 Gene Therapy Market - Regional Analysis

18.2.1 US

18.2.2 Europe

18.2.3 Japan

18.2.4 China

18.2.5 India

18.2.6 South Korea

18.2.7 Taiwan

19. Mergers & Collaborations between Major Key Players of the Market

19.1 Axovant Gene Therapies & Yposkesi's Strategic Partnership

19.2 Lonza and DiNAQOR AG's Strategic Collaboration

19.3 US$ 1 Million to AavantiBio for Gene Therapy Development in Freidreich's Ataxia

19.4 Actinium Pharmaceuticals Collaboration with UC Davis for HIV-Related Lymphoma for ACT Gene Therapy Program

20. Global Gene Therapy Market Dynamics

20.1 Factors Driving the Growth of Gene Therapy

20.1.1 Gene Therapy Dominant over Growing Prevalence of Cancer

20.1.2 Increased R&D Activities with Rising Investments

20.1.3 Increasing General Awareness Concerning Gene Therapy

20.2 Challenges Overpowering the Technology

20.3 Future Opportunities for Gene Therapy

21. Competitive Landscape

21.1 Novartis

21.2 Spark Therapeutics

21.3 BioGen

21.4 Sarepta Therapeutics

21.5 Kite Pharma

21.6 Amgen

21.7 Solid Biosciences Inc.

21.8 uniQure NV

21.9 MeiraGTX Holdings

21.10 Audentis Therapeutics

21.11 Regenxbio Inc.

21.12 Alnylam Pharmaceuticals

21.13 Arrowhead

21.14 SQZ Biotechnologies

21.15 Bluerock Therapeutics

21.16 Zydus Takeda

21.17 Intrexon Corporation

21.18 Celgene

21.19 Roche

21.20 Oxford Biomedica

21.21 Genethon

21.22 Sangamo Biosciences

21.23 Juno Therapeutics

21.24 Cellectis

21.25 Autolus Therapeutics plc

21.26 icell Gene Therapeutics

21.27 Allogene Therapeutics

For more information about this report visit

Laura Wood, Senior Press Manager
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900

Laura Wood, Senior Press Manager
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900