StemCell Therapy

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.

The research reports onEmerging Gene Therapies Marketreport 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.

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Emerging Gene Therapies Market Top Company Analysis-Sangamo TherapeuticsCRISPR TherapeuticsCasebia TherapeuticsEditas MedicineIntellia TherapeuticsHomology Medicines

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Emerging Gene Therapies Market 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 Emerging Gene Therapies Market 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?

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Reasons to accessthis 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.

Table of Contents in this Report-1 Table of Contents 51.1 List of Tables 71.2 List of Figures 82 Introduction 102.1 Gene Therapy Definitions 102.2 Report Coverage the Emerging Gene Therapy Pipeline 112.3 History of Gene Therapy 122.4 Limitations of Gene Transfer 132.5 The Development of Targeted Gene Editing 132.6 Overview of Gene Editing Platforms 132.6.1 Zinc Fingers (1996) 132.6.2 Transcription Activator-Like Effectors (2011) 142.6.3 The CRISPR/Cas System (2013) 152.6.4 Effectors for Targeting Domains 192.6.5 Comparison of Gene Editing Systems 192.6.6 Summary of Gene Editing Systems 192.7 Overview of In Vivo Gene Therapy 212.7.1 Editing is Dependent on Cell Type, Stage, and Repair Pathway 212.7.2 Delivery 212.7.3 Emerging Safety Concerns with Editing Platforms 242.7.4 Editing Products are Reliant on the Target Cells Cycle Stage and DNA Repair Machinery 272.7.5 Advantages of Gene Editing over Gene Transfer 282.7.6 Integration into Safe Harbor Sites 282.7.7 The Increasing Complexity of Gene Therapy 302.7.8 Summary of In Vivo Gene Therapy 313 Gene Therapy Near Term Product Pipeline 333.1 Leber Congenital Amaurosis 333.1.1 Unmet Need 333.1.2 Molecular Genetics 333.1.3 Luxturna (Voretigene neparvovec) 333.1.4 Editas Medicine: EDIT-101 353.1.5 Trial Design 363.1.6 EDIT-101 and Off-Target Effects 373.1.7 The Potential Advantage of EDIT-101 is the Longevity of its Therapeutic Effect 373.1.8 Summary LCA 383.2 Choroideremia 383.3 Hurler Syndrome (MPS I) 393.3.1 Key Clinical Studies 403.3.2 Regenex: RGX-111 403.3.3 Sangamo Therapeutics: SB-318 403.4 Hunter Syndrome (MPS II) 413.4.1 Unmet Need 413.4.2 Sangamo Therapeutics: SB-913 413.4.3 Immusoft Corporation: Cell Therapy 433.5 Sanfilippo Syndrome (MPS III) 433.5.1 Lysogene: LYS-SAF302 433.6 Summary MPS Disorders 443.7 Hemophilia 443.7.1 Hemophilia A 463.7.2 Summary Hemophilia A 503.7.3 Hemophilia B 513.7.4 Summary Hemophilia B 533.8 Hemoglobinopathies 543.8.1 Beta Thalassemia: Unmet Need 543.8.2 Beta Thalassemia: Molecular Genetics 553.8.3 Sickle Cell Disease: Unmet Need 563.8.4 Sickle Cell Disease: Molecular Genetics 563.9 Cellular Therapies for Hemoglobinopathies 573.9.1 Blue Bird Bio: BB-305 (LentiGlobin) 573.9.2 Sangamo: ST-400 603.9.3 CRISPR Therapeutics: CTX-001 613.9.4 Summary: Cellular Therapies for Hemoglobinopathies 623.10 Duchenne Muscular Dystrophy 633.10.1 Unmet Need 633.10.2 Molecular Genetics 633.10.3 ExonDys 51 Sarepta Therapeutics 643.10.4 Solid BioSciences: SGT-001 663.10.5 Exonics Therapeutics: CRISPR Approach 673.10.6 Summary Duchenne Muscular Dystrophy 684 Competitive Landscape 694.1 Regulatory Considerations for Developing Gene Therapy Products 694.1.1 Product Characteristics 694.1.2 Clinical Study Design for Gene Therapy Products 694.1.3 Disease specific guidance 704.1.4 Reimbursement and Payment 714.1.5 Summary Regulatory Considerations 724.2 Intellectual Property CRISPR/Cas 724.2.1 Licensing, Exploitation, and MPEG Pool 744.3 Company Analysis: Gene Editing Companies 754.3.1 Sangamo Therapeutics 754.3.2 CRISPR Therapeutics 794.3.3 Casebia Therapeutics 814.3.4 Editas Medicine 824.3.5 Intellia Therapeutics 844.3.6 Homology Medicines 864.4 Company Analysis: Pharma 874.4.1 Amgen 874.4.2 Gilead Sciences 874.4.3 Novartis 874.4.4 Sanofi 884.4.5 GlaxoSmithKline 884.4.6 Pfizer 885 Appendix 895.1 References 895.2 Report Methodology 98

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Current research: 2020 Emerging Gene Therapies Market Report - WhaTech Technology and Markets News

This entry was posted on July 9, 2020 at 6:43 pm and is filed under Molecular Genetics. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |