Presently, there are more than 10 approved gene therapies; over 465 product candidates are being evaluated
Encouragingclinical results across various metabolic, hematological and ophthalmicdisorders have inspired research groups across the world to focus their effortson the development of novel gene editing therapies. In fact, the gene therapy pipelinehas evolved significantly over the past few years, with three products beingapproved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA® (AveXis)and ZYNTEGLO™ (bluebird bio). Further, there are multiple pipeline candidates inmid to late-stage (phase II and above) trials that are anticipated to enter themarket over the next 5-10 years.
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The USD 11.6 billion (by 2030) financialopportunity within the gene therapy market has been analyzed across thefollowing segments:
§ Key therapeutic areas
§ Autoimmune disorders
§ Cardiovascular diseases
§ Genetic disorders
§ Hematologicaldisorders
§ Metabolic disorders
§ Ophthalmic disorders
§ Oncological disorders
§ Others
§ Type of vector
§ Adeno associatedvirus
§ Adenovirus
§ Herpes simplex virustype 1
§ Lentivirus
§ Plasmid DNA
§ Retrovirus
§ Vaccinia Virus
§ Type of therapy
§ Exvivo
§ In vivo
§ Type of genemodification
§ Gene augmentation
§ Immunotherapy
§ Oncolytic therapy
§ Others
§ Route ofadministration
§ Intraarticular
§ Intracerebellar
§ Intramuscular
§ Intradermal
§ Intravenous
§ Intravitreal
§ Intravesical
§ Subretinal
§ Others
§ Key geographicalregions
§ North America
§ Europe
§ Asia-Pacific
The Gene Therapy Market (3rd Edition), 2019-2030 reportfeatures the following companies, which we identified to be key players in thisdomain:
§ Advantagene
§ Advaxis
§ BioMarin
§ bluebird bio
§ FKD Therapies
§ Freeline Therapeutics
§ GenSight Biologics
§ Gradalis
§ InovioPharmaceuticals
§ 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 ReimbursementScenario
6. Competitive Landscape
7. Marketed Gene Therapies
8. Key Commercialization Strategies
9. Late Stage (Phase II/III and Above) GeneTherapies
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 GeneTherapy 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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ContactDetails
GauravChaudhary
+1(415) 800 3415
gaurav.chaudhary@rootsanalysis.com
