Friday, 15 February 2019

Emerging Gene Therapies - Trends within the Technological, Clinical, Regulatory and Competitive Landscape

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Latest report Emerging Gene Therapies - Trends within the Technological, Clinical, Regulatory and Competitive Landscape 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.

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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

- 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

- 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.

Table of Contents

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 Cells 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

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