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
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Contact Details
Gaurav Chaudhary
+1 (415) 800 3415
gaurav.chaudhary@rootsanalysis.com