Presently, there are more than 10 approved gene therapies / Roots Analysis

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA® (AveXis) and ZYNTEGLO™ (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

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The USD 11.6 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

§  Key therapeutic areas

§  Autoimmune disorders

§  Cardiovascular diseases

§  Genetic disorders

§  Hematological disorders

§  Metabolic disorders

§  Ophthalmic disorders

§  Oncological disorders

§  Others

§  Type of vector

§  Adeno associated virus

§  Adenovirus

§  Herpes simplex virus type 1

§  Lentivirus

§  Plasmid DNA

§  Retrovirus

§  Vaccinia Virus

§  Type of therapy

§  Ex vivo

§  In vivo

§  Type of gene modification

§  Gene augmentation

§  Immunotherapy

§  Oncolytic therapy

§  Others

§  Route of administration

§  Intraarticular

§  Intracerebellar

§  Intramuscular

§  Intradermal

§  Intravenous

§  Intravitreal

§  Intravesical

§  Subretinal

§  Others

§  Key geographical regions

§  North America

§  Europe

§  Asia-Pacific

The Gene Therapy Market (3rd Edition), 2019-2030 report features the following companies, which we identified to be key players in this domain:

§  Advantagene

§  Advaxis

§  BioMarin

§  bluebird bio

§  FKD Therapies

§  Freeline Therapeutics

§  GenSight Biologics

§  Gradalis

§  Inovio Pharmaceuticals

§  Marsala Biotech

§  Orchard Therapeutics

§  Pfizer

§  Sarepta Therapeutics

§  Spark Therapeutics

§  Tocagen

§  Transgene

§  uniQure Biopharma

§  VBL Therapeutics

§  ViroMed

Table of Contents

1. Preface

2. Executive Summary

3. Introduction

4. Gene Delivery Vectors

5. Regulatory Landscape and Reimbursement Scenario

6. Competitive Landscape

7. Marketed Gene Therapies

8. Key Commercialization Strategies

9. Late Stage (Phase II/III and Above) Gene Therapies

10. Emerging Technologies

11. Promising Therapeutics Areas

12. Patent Analysis

13. Mergers and Acquisitions

14. Funding and Investment Analysis

15. Cost Price Analysis

16. Big Pharma Players: Analysis of Gene Therapy Related Initiatives

17. Market Forecast and Opportunity Analysis

18. Vector Manufacturing

19. Case Study: Gene Therapy Supply Chain

20. Conclusion

21. Interview Transcripts

22. Appendix 1: Tabulated Data

23. Appendix 2: List of Companies and Organizations

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Contact Details

Gaurav Chaudhary

+1 (415) 800 3415

gaurav.chaudhary@rootsanalysis.com