Home > > > Report Detail

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

Publisher Name :
Date: 03-Jan-2019
No. of pages: 99

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

Summary

GBI Research's 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.

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.

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

1 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 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
5.3 About GBI Research 99
5.4 Disclaimer 99

1.1 List of Tables
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

1.2 List of Figures
Figure 1: Nucleases Based on Protein-DNA Interactions 14
Figure 2: Transcription Activator-Like Effector Nucleases (TALENs) 15
Figure 3: The CRISPR/Cas System 16
Figure 4: CRISPR/Cas Binding Mechanism 17
Figure 5: Cas9 Orthologs 18
Figure 6: Gene Therapy Clinical Trials Worldwide by Vector 22
Figure 7: Multiple Sangamo Therapeutics Products Use an Albumin Targeting ‘Safe Harbor' Approach 29
Figure 8: Gene Editing Landscape, Progression of Gene Therapy Applications 30
Figure 9: Gene Editing Landscape, Hemophilia A + B 44
Figure 10: SPK-9001: Factor IX Activity after SPK-9011 in 8 Participants that did not Show AAV Capsid-Directed Immune Response 53
Figure 11: Cost of Beta-Thalassemia Major Treatment as of NHS Tariffs at 2013/14 Prices 55
Figure 12: BCL11A is Involved in Fetal Hemoglobin Silencing 60
  • Global Antioxidant Vitamins Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 100
    The global Antioxidant Vitamins market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Antioxidant Vitamins is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Antioxidant Vitamins is estimated to increase from $ million in 2023 to reach $ millio......
  • Global Clavulanate Potassium Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 86
    The global Clavulanate Potassium market was valued at US$ 446.6 million in 2023 and is anticipated to reach US$ 637.2 million by 2030, witnessing a CAGR of 5.2% during The forecast period 2024-2030. North American market for Clavulanate Potassium is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Clavulanate Potassium is estimated to increase from $ million in 2023......
  • Global Amoxicillin Sodium Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 84
    The global Amoxicillin Sodium market was valued at US$ 460.9 million in 2023 and is anticipated to reach US$ 735.1 million by 2030, witnessing a CAGR of 7.0% during The forecast period 2024-2030. North American market for Amoxicillin Sodium is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Amoxicillin Sodium is estimated to increase from $ million in 2023 to reach......
  • Global Melatonin Supplements Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 106
    The global Melatonin Supplements market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Melatonin Supplements is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Melatonin Supplements is estimated to increase from $ million in 2023 to reach $ mil......
  • Global Sleeping Pills (Prescription Drugs) Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 93
    The global Sleeping Pills (Prescription Drugs) market was valued at US$ 1022.3 million in 2023 and is anticipated to reach US$ 1437.8 million by 2030, witnessing a CAGR of 4.9% during The forecast period 2024-2030. The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical......
  • Global Sleeping Pills Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 94
    The global Sleeping Pills market was valued at US$ 2042.3 million in 2023 and is anticipated to reach US$ 2979.5 million by 2030, witnessing a CAGR of 3.8% during The forecast period 2024-2030. The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical drug market is estim......
  • Global Zinc Oral Drops Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 85
    The global Zinc Oral Drops market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Zinc Oral Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Zinc Oral Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a......
  • Global Zinc Drops Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 85
    The global Zinc Drops market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030. North American market for Zinc Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030. Asia-Pacific market for Zinc Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % duri......
  • Global Anti-Infectives Market Research Report 2024
    Published: 10-Jan-2024        Price: US 2900 Onwards        Pages: 78
    The global Anti-Infectives market was valued at US$ 22700 million in 2023 and is anticipated to reach US$ 27480 million by 2030, witnessing a CAGR of 2.3% during The forecast period 2024-2030. The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical drug market is estima......
    • SERVICES
    Value for Money
    We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
    Ever Growing Inventory
    Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
    One Stop Solution
    Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
    Dedicated Client Engagement
    Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
    Saving Time and Efforts
    Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
    Payment Flexibility
    Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
    Post-Purchase Research Support
    Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements. Subscription Offers & Packages (Get in touch with us for more details - [email protected] / +1 888 391 5441 )
    • Ad Hoc
    • Pay - as - you - go / Bucket Subscriptions
    • Fixed Cost for #of reports
    • Customize / Personalize as per your needs