Emerging Gene Therapies Market Report- Current Challenges, Top Pharmaceutical Companies and Regulatory Trends

The research reports on Emerging Gene Therapies Market report gives detailed overview of factors that affect global business scope. Emerging Gene Therapies Market report shows the latest market insights with upcoming trends and breakdowns of products and services. This report provides statistics on the market situation, size, regions and growth factors. Emerging Gene Therapies Market report contains emerging players analyze data including competitive situations, sales, revenue and market share of top manufacturers.

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Emerging Gene Therapies Market Report including Top Companies-
Sangamo Therapeutics
CRISPR Therapeutics
Casebia Therapeutics
Editas Medicine
Intellia Therapeutics
Homology Medicines
and more...

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.

Additionally, the 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.

The 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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Table of Contents in this Report-
1 Table of Contents 5
1.1 List of Tables 7
1.2 List of Figures 8
2 Introduction 10
2.1 Gene Therapy - Definitions 10
2.2 Report Coverage - the Emerging Gene Therapy Pipeline 11
2.3 History of Gene Therapy 12
2.4 Limitations of Gene Transfer 13
2.5 The Development of Targeted Gene Editing 13
2.6 Overview of Gene Editing Platforms 13
2.6.1 Zinc Fingers (1996) 13
2.6.2 Transcription Activator-Like Effectors (2011) 14
2.6.3 The CRISPR/Cas System (2013) 15
2.6.4 Effectors for Targeting Domains 19
2.6.5 Comparison of Gene Editing Systems 19
2.6.6 Summary of Gene Editing Systems 19
2.7 Overview of In Vivo Gene Therapy 21
2.7.1 Editing is Dependent on Cell Type, Stage, and Repair Pathway 21
2.7.2 Delivery 21
2.7.3 Emerging Safety Concerns with Editing Platforms 24
2.7.4 Editing Products are Reliant on the Target Cell’s Cycle Stage and DNA Repair Machinery 27
2.7.5 Advantages of Gene Editing over Gene Transfer 28
2.7.6 Integration into ‘Safe Harbor’ Sites 28
2.7.7 The Increasing Complexity of Gene Therapy 30
2.7.8 Summary of In Vivo Gene Therapy 31
3 Gene Therapy - Near Term Product Pipeline 33
3.1 Leber Congenital Amaurosis 33
3.1.1 Unmet Need 33
3.1.2 Molecular Genetics 33
3.1.3 Luxturna (Voretigene neparvovec) 33
3.1.4 Editas Medicine: EDIT-101 35
3.1.5 Trial Design 36
3.1.6 EDIT-101 and Off-Target Effects 37
3.1.7 The Potential Advantage of EDIT-101 is the Longevity of its Therapeutic Effect 37
3.1.8 Summary - LCA 38
3.2 Choroideremia 38
3.3 Hurler Syndrome (MPS I) 39
3.3.1 Key Clinical Studies 40
3.3.2 Regenex: RGX-111 40
3.3.3 Sangamo Therapeutics: SB-318 40
3.4 Hunter Syndrome (MPS II) 41
3.4.1 Unmet Need 41
3.4.2 Sangamo Therapeutics: SB-913 41
3.4.3 Immusoft Corporation: Cell Therapy 43
3.5 Sanfilippo Syndrome (MPS III) 43
3.5.1 Lysogene: LYS-SAF302 43
3.6 Summary - MPS Disorders 44
3.7 Hemophilia 44
3.7.1 Hemophilia A 46
3.7.2 Summary - Hemophilia A 50
3.7.3 Hemophilia B 51
3.7.4 Summary - Hemophilia B 53
3.8 Hemoglobinopathies 54
3.8.1 Beta Thalassemia: Unmet Need 54
3.8.2 Beta Thalassemia: Molecular Genetics 55
3.8.3 Sickle Cell Disease: Unmet Need 56
3.8.4 Sickle Cell Disease: Molecular Genetics 56
3.9 Cellular Therapies for Hemoglobinopathies 57
3.9.1 Blue Bird Bio: BB-305 (‘LentiGlobin’) 57
3.9.2 Sangamo: ST-400 60
3.9.3 CRISPR Therapeutics: CTX-001 61
3.9.4 Summary: Cellular Therapies for Hemoglobinopathies 62
3.10 Duchenne Muscular Dystrophy 63
3.10.1 Unmet Need 63
3.10.2 Molecular Genetics 63
3.10.3 ExonDys 51 - Sarepta Therapeutics 64
3.10.4 Solid BioSciences: SGT-001 66
3.10.5 Exonics Therapeutics: CRISPR Approach 67
3.10.6 Summary - Duchenne Muscular Dystrophy 68
4 Competitive Landscape 69
4.1 Regulatory Considerations for Developing Gene Therapy Products 69
4.1.1 Product Characteristics 69
4.1.2 Clinical Study Design for Gene Therapy Products 69
4.1.3 Disease specific guidance 70
4.1.4 Reimbursement and Payment 71
4.1.5 Summary - Regulatory Considerations 72
4.2 Intellectual Property - CRISPR/Cas 72
4.2.1 Licensing, Exploitation, and MPEG Pool 74
4.3 Company Analysis: Gene Editing Companies 75
4.3.1 Sangamo Therapeutics 75
4.3.2 CRISPR Therapeutics 79
4.3.3 Casebia Therapeutics 81
4.3.4 Editas Medicine 82
4.3.5 Intellia Therapeutics 84
4.3.6 Homology Medicines 86
4.4 Company Analysis: Pharma 87
4.4.1 Amgen 87
4.4.2 Gilead Sciences 87
4.4.3 Novartis 87
4.4.4 Sanofi 88
4.4.5 GlaxoSmithKline 88
4.4.6 Pfizer 88
5 Appendix 89
5.1 References 89
5.2 Report Methodology 98
and more...

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This Report provides detailed knowledge of upcoming market trends and current conditions in the global market. This report covers the past, present and forecast period for the long-term and collective examination of the Keyword.

This release was published on openPR.