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

Author : kevin987
Publish Date : 2021-03-23 05:23:52

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.

Key Inclusions

  • A detailed review of the overall landscape of gene therapies and genome editing therapies, including information on various drug / therapy developer companies, phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others) and target disease indication(s), information on gene type, type of vector used, type of therapy (ex vivo and in vivo), mechanism of action, type of gene modification (gene augmentation, oncolytic viral therapy and others) and special drug designation (if any).
  • A discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vectors.
  • A world map representation, depicting the most active geographies, in terms of the presence of companies engaged in developing gene therapies, and a bull's eye analysis, highlighting the distribution of clinical-stage pipeline candidates by phase of development, type of vector and type of therapy (ex vivo and in vivo).
  • A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.

Detailed profiles of marketed and phase II/III and gene therapies, including a brief history of development, information on current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, along with information on the developer company


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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


To purchase a copy, please visit


Contact Details

Gaurav Chaudhary

+1 (415) 800 3415

[email protected]

Category : general

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