Emerging Gene Therapies Market Report- Trends within the Technological and Competitive Landscape: Sanofi, GlaxoSmithKline, Pfizer

RNR has published a report on Emerging Gene Therapies Market such that the entire market dynamics and analytics are well represented through the various segments such as product type, application, end-user, regions, and dominant players in the market. Emerging Gene Therapies Market report also covers all the market valuation (share, size, and revenue) coupled with complete overview. Emerging Gene Therapies Market in-depth market analysis is likely to enlighten the readers and the investors with the necessary strategic tactics, growth drivers, restraints, opportunities, profits, pricing, and market growth phase for a better understanding of the Emerging Gene Therapies Market.

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Top Company Profile Analysis in this Report-
Sangamo Therapeutics
CRISPR Therapeutics
Casebia Therapeutics
Editas Medicine
Intellia Therapeutics
Homology Medicines
Company Analysis: Pharma
Amgen
Gilead Sciences
Novartis
Sanofi
GlaxoSmithKline
Pfizer

Emerging Gene Therapies Market Report provides a comprehensive overview of the emerging gene therapy market. The report discusses gene therapy and the technology behind gene editing, outlining the advantages, limitations and current evidence for the platforms under development. The report discusses relevant clinical studies targeting specific therapeutic indications and highlights examples of current challenges within the field, with a focus on therapies that target the eye, liver, and blood.

Emerging Gene Therapies Market Report provides a background to the CRISPR patent litigation, a key factor within the gene editing company landscape. It provides profiles of six companies developing gene editing platforms, considers the gene therapy interests of the main pharmaceutical companies, and discusses current regulatory trends in the development of gene therapies.

Emerging Gene Therapies Market Report explores how emerging gene editing products will compete with established products, their relative competitive strengths, and upcoming value inflection points within the field.

Scope of this Report-
- What are the key emerging products within the gene therapy landscape?
- Which companies have the strongest pipeline of innovative products?
- How will gene editing disrupt existing gene therapy products?
- What are the regulatory trends for emerging gene therapies?
- What are the interests of pharmaceutical companies within the field?

Reasons to buy this Report-
- Achieve an up-to-date understanding of the area, with a comprehensive reference of key products within the gene therapy landscape, compared across technology-specific relevant characteristics such as editing mechanism and delivery vector.
- Conduct competitive analysis using indication-specific, side-by-side comparisons of the latest data for key gene therapy products in the strategically relevant areas of eye, blood, and liver.
- Conduct strategic analysis using an overview of gene therapy specific considerations for evaluating and developing gene therapy products - the CRISPR patent space, emerging regulatory trends, innovation leaders and the interests of pharma in gene therapy.

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List of Tables in this Report-
Table 1: Pipeline Products Covered 13
Table 2: Properties of Standard Gene-Editing Nucleases 20
Table 3: Registered Clinical Trials of CAR-T Cell Therapies Using Gene-Editing 21
Table 4: Gene Therapy Clinical Trials Worldwide by Vector 24
Table 5: Gene Editing Landscape, Vector Differentiation by Target Tissue 25
Table 6: Gene Editing Landscape, Key Off-Target Effect Studies 26
Table 7: Pre-existing Immunity to Cas9 27
Table 8: Characterization of the Rate of Homology Directed Repair in Range of Cell Lines 28
Table 9: Comparison of Gene Editing and Gene Transfer Approaches 29
Table 10: Gene Editing Landscape, Pipeline of Targeted Gene Editing Products by Company 32
Table 11: Luxturna Clinical Studies 35
Table 12: EDIT-101 - Phase I/II Trial Design and Comparison to Luxturna Efficacy Study 38
Table 13: Dose Response in CEP290 Gene Editing and CRISPR Expression 39
Table 14: Gene editing landscape, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), Diagnosed, Prevalent Cases, 2017-2027 40
Table 15: Key Phase I/II Studies in MPS I 41
Table 16: Gene editing landscape, Mucopolysaccharidosis II (MPS II) (Hunter Syndrome), Diagnosed, Prevalent Cases, 2017-2027. 42
Table 17: SB-913 Trial Design 43
Table 18: SB-913 - Interim Results 43
Table 19: LYS-SAF302 Phase II/III Trial Design 44
Table 20: Gene Editing Landscape, MPS Disorders 45
Table 21: Gene Editing Landscape, Hemophilia Epidemiology and Forecast, 2016-2026. 46
Table 22: Scale of Hemophilia Severity 47
Table 23: Phase I/II Hemophilia A Trials 48
Table 24: BMN-270 Factor VIII Levels at 1.5 Years (High Dose) 49
Table 25: Gene Therapy Landscape, Phase I/II Clinical Trial Design for Hemophilia A 50
Table 26: Gene Therapy Landscape, Summary of Key Pipeline Gene Therapies for Hemophilia A 51
Table 27: Phase I/II Clinical Studies 52
Table 28: Gene Therapy Landscape, Overview Of Key Pipeline Products in Hemophilia B 54
Table 29: Gene editing landscape, Thalassemia, Diagnosed, Prevalent Cases, 2017-2027 55
Table 30: Frequent Mutations Causing SCD 57
Table 31: Phase I/II Clinical Studies 58
Table 32: LentiGlobin - Ongoing Phase III Trials in Beta Thalassemia 59
Table 33: Results of LentiGlobin trials in Thalassemia and Sickle Cell Disease 60
Table 34: ST-400 Trial Design Compared with LentiGlobin 62
Table 35: CTX-001 trial design (Left) against ST-400 and LentiGlobin (Right) 63
Table 36: LentiGlobin (BB-305) may Achieve Blockbuster Status by 2023 64
Table 37: Gene Therapy Landscape, DMD, Global Prevalence (%) 64
Table 38: Exondys 51 - Clinical Studies 66
Table 39: SGT- 001 Study Design 67
Table 40: Gene Therapy Landscape, Key Patents in the CRISPR Dispute 74
Table 41: Gene Therapy Landscape, IP Estates of CRISPR Companies 75
Table 42: Gene Therapy Landscape, ERS Genomics EU Licensing 2004-2018 76
Table 43: Sangamo Therapeutics Pipeline, September 2018 76
Table 44: Sangamo Therapeutics Partnerships 78
Table 45: Sangamo Therapeutics SWOT 79
Table 46: CRISPR Therapeutics Pipeline 80
Table 47: CRISPR Therapeutics Pipeline 81
Table 48: CRISPR Therapeutics SWOT 82
Table 49: Casebia Pipeline, September 2018 82
Table 50: Editas Medicine Pipeline, September 2018 83
Table 51: Editas Medicine Pipeline, September 2018. 84
Table 52: Editas Medicine SWOT 85
Table 53: Intellia Therapeutics Pipeline 85
Table 54: Intellia Therapeutics SWOT 86
Table 55: Homology Medicines Pipeline 87
Table 56: Homology Medicines SWOT 88
and more..

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