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

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

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


Genomic surgery is increasingly a reality. The last two years have seen the approval of four gene therapies by the FDA - (1) Luxturna, the first in vivo gene transfer therapy (2) Imlygic, an novel immunotherapy, (3) Kymriah and (4) Yescarta, two Chimeric Antigen Receptor T - Cell therapies targeting CD19 for oncological conditions. Over the next two years, a number of key events will determine the clinical tractability and commercial interest of gene editing approaches. These will be pivotal within the gene therapy field.

Until recently, the field has had limited success outside of CAR-T therapies. Ex vivo cell therapy Strimvelis for ADA-SCID was approved in 2016, but has subsequently been sold by its developer GSK, due to lack of profitability. The first exon skipping therapy, ExonDys 51, for Duchenne Muscular Dystrophy was approved controversially by the FDA in 2016, but failed to gain approval in Europe based on its efficacy, and its US sales are reportedly slow.

A key challenge in gene therapy development has been delivering enough product into the target tissue. This has meant many gene therapy products have struggled to achieve a high enough level of therapeutic gene expression to induce a clinical effect. Naturally, selection of therapy areas for the development of novel gene therapy products has been influenced by these technical challenges. Drug developers have prioritized conditions with well-understood pathology, those with simple genetics that are able to be corrected using simpler editing approaches, and those in which the delivery problem is minimized.

Due to the relative ease of achieving high levels of gene therapy product to cells ex vivo, development has been focused in areas where cellular therapy is an established part of clinical procedure - for instance, in the hemoglobinopathies (e.g. beta thalassemia, sickle cell disease) and the lysosomal disorders, where hematopoietic stem cell transfer is commonly used as part of clinical care. Ex vivo products have also seen success in oncology, where ex vivo modification of T-Cells has to express antigens as a method for immunotherapy has achieved remarkable clinical results.

The report Emerging Gene Therapies - Trends within the Technological, Clinical, Regulatory and Competitive Landscape provides a comprehensive overview of the emerging gene therapy market. It 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.


  • 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.

1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Introduction
2.1 Gene Therapy - Definitions
2.2 Report Coverage - the Emerging Gene Therapy Pipeline
2.3 History of Gene Therapy
2.4 Limitations of Gene Transfer
2.5 The Development of Targeted Gene Editing
2.6 Overview of Gene Editing Platforms
2.6.1 Zinc Fingers (1996)
2.6.2 Transcription Activator-Like Effectors (2011)
2.6.3 The CRISPR/Cas System (2013)
2.6.4 Effectors for Targeting Domains
2.6.5 Comparison of Gene Editing Systems
2.6.6 Summary of Gene Editing Systems
2.7 Overview of In Vivo Gene Therapy
2.7.1 Editing is Dependent on Cell Type, Stage, and Repair Pathway
2.7.2 Delivery
2.7.3 Emerging Safety Concerns with Editing Platforms
2.7.4 Editing Products are Reliant on the Target Cell’s Cycle Stage and DNA Repair Machinery
2.7.5 Advantages of Gene Editing over Gene Transfer
2.7.6 Integration into ‘Safe Harbor’ Sites
2.7.7 The Increasing Complexity of Gene Therapy
2.7.8 Summary of In Vivo Gene Therapy
3 Gene Therapy - Near Term Product Pipeline
3.1 Leber Congenital Amaurosis
3.1.1 Unmet Need
3.1.2 Molecular Genetics
3.1.3 Luxturna (Voretigene neparvovec)
3.1.4 Editas Medicine: EDIT-101
3.1.5 Trial Design
3.1.6 EDIT-101 and Off-Target Effects
3.1.7 The Potential Advantage of EDIT-101 is the Longevity of its Therapeutic Effect
3.1.8 Summary - LCA
3.2 Choroideremia
3.3 Hurler Syndrome (MPS I)
3.3.1 Key Clinical Studies
3.3.2 Regenex: RGX-111
3.3.3 Sangamo Therapeutics: SB-318
3.4 Hunter Syndrome (MPS II)
3.4.1 Unmet Need
3.4.2 Sangamo Therapeutics: SB-913
3.4.3 Immusoft Corporation: Cell Therapy
3.5 Sanfilippo Syndrome (MPS III)
3.5.1 Lysogene: LYS-SAF302
3.6 Summary - MPS Disorders
3.7 Hemophilia
3.7.1 Hemophilia A
3.7.2 Summary - Hemophilia A
3.7.3 Hemophilia B
3.7.4 Summary - Hemophilia B
3.8 Hemoglobinopathies
3.8.1 Beta Thalassemia: Unmet Need
3.8.2 Beta Thalassemia: Molecular Genetics
3.8.3 Sickle Cell Disease: Unmet Need
3.8.4 Sickle Cell Disease: Molecular Genetics
3.9 Cellular Therapies for Hemoglobinopathies
3.9.1 Blue Bird Bio: BB-305 (‘LentiGlobin’)
3.9.2 Sangamo: ST-400
3.9.3 CRISPR Therapeutics: CTX-001
3.9.4 Summary: Cellular Therapies for Hemoglobinopathies
3.10 Duchenne Muscular Dystrophy
3.10.1 Unmet Need
3.10.2 Molecular Genetics
3.10.3 ExonDys 51 - Sarepta Therapeutics
3.10.4 Solid BioSciences: SGT-001
3.10.5 Exonics Therapeutics: CRISPR Approach
3.10.6 Summary - Duchenne Muscular Dystrophy
4 Competitive Landscape
4.1 Regulatory Considerations for Developing Gene Therapy Products
4.1.1 Product Characteristics
4.1.2 Clinical Study Design for Gene Therapy Products
4.1.3 Disease specific guidance
4.1.4 Reimbursement and Payment
4.1.5 Summary - Regulatory Considerations
4.2 Intellectual Property - CRISPR/Cas
4.2.1 Licensing, Exploitation, and MPEG Pool
4.3 Company Analysis: Gene Editing Companies
4.3.1 Sangamo Therapeutics
4.3.2 CRISPR Therapeutics
4.3.3 Casebia Therapeutics
4.3.4 Editas Medicine
4.3.5 Intellia Therapeutics
4.3.6 Homology Medicines
4.4 Company Analysis: Pharma
4.4.1 Amgen
4.4.2 Gilead Sciences
4.4.3 Novartis
4.4.4 Sanofi
4.4.5 GlaxoSmithKline
4.4.6 Pfizer
5 Appendix
5.1 References
5.2 Report Methodology
5.3 About GBI Research
5.4 Disclaimer
1.1 List of Tables
Table 1: Pipeline Products Covered
Table 2: Properties of Standard Gene-Editing Nucleases
Table 3: Registered Clinical Trials of CAR-T Cell Therapies Using Gene-Editing
Table 4: Gene Therapy Clinical Trials Worldwide by Vector
Table 5: Gene Editing Landscape, Vector Differentiation by Target Tissue
Table 6: Gene Editing Landscape, Key Off-Target Effect Studies
Table 7: Pre-existing Immunity to Cas9
Table 8: Characterization of the Rate of Homology Directed Repair in Range of Cell Lines
Table 9: Comparison of Gene Editing and Gene Transfer Approaches
Table 10: Gene Editing Landscape, Pipeline of Targeted Gene Editing Products by Company
Table 11: Luxturna Clinical Studies
Table 12: EDIT-101 - Phase I/II Trial Design and Comparison to Luxturna Efficacy Study
Table 13: Dose Response in CEP290 Gene Editing and CRISPR Expression
Table 14: Gene editing landscape, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), Diagnosed, Prevalent Cases, 2017-2027
Table 15: Key Phase I/II Studies in MPS I
Table 16: Gene editing landscape, Mucopolysaccharidosis II (MPS II) (Hunter Syndrome), Diagnosed, Prevalent Cases, 2017-2027.
Table 17: SB-913 Trial Design
Table 18: SB-913 - Interim Results
Table 19: LYS-SAF302 Phase II/III Trial Design
Table 20: Gene Editing Landscape, MPS Disorders
Table 21: Gene Editing Landscape, Hemophilia Epidemiology and Forecast, 2016-2026.
Table 22: Scale of Hemophilia Severity
Table 23: Phase I/II Hemophilia A Trials
Table 24: BMN-270 Factor VIII Levels at 1.5 Years (High Dose)
Table 25: Gene Therapy Landscape, Phase I/II Clinical Trial Design for Hemophilia A
Table 26: Gene Therapy Landscape, Summary of Key Pipeline Gene Therapies for Hemophilia A
Table 27: Phase I/II Clinical Studies
Table 28: Gene Therapy Landscape, Overview Of Key Pipeline Products in Hemophilia B
Table 29: Gene editing landscape, Thalassemia, Diagnosed, Prevalent Cases, 2017-2027
Table 30: Frequent Mutations Causing SCD
Table 31: Phase I/II Clinical Studies
Table 32: LentiGlobin - Ongoing Phase III Trials in Beta Thalassemia
Table 33: Results of LentiGlobin trials in Thalassemia and Sickle Cell Disease
Table 34: ST-400 Trial Design Compared with LentiGlobin
Table 35: CTX-001 trial design (Left) against ST-400 and LentiGlobin (Right)
Table 36: LentiGlobin (BB-305) may Achieve Blockbuster Status by 2023
Table 37: Gene Therapy Landscape, DMD, Global Prevalence (%)
Table 38: Exondys 51 - Clinical Studies
Table 39: SGT- 001 Study Design
Table 40: Gene Therapy Landscape, Key Patents in the CRISPR Dispute
Table 41: Gene Therapy Landscape, IP Estates of CRISPR Companies
Table 42: Gene Therapy Landscape, ERS Genomics EU Licensing 2004-2018
Table 43: Sangamo Therapeutics Pipeline, September 2018
Table 44: Sangamo Therapeutics Partnerships
Table 45: Sangamo Therapeutics SWOT
Table 46: CRISPR Therapeutics Pipeline
Table 47: CRISPR Therapeutics Pipeline
Table 48: CRISPR Therapeutics SWOT
Table 49: Casebia Pipeline, September 2018
Table 50: Editas Medicine Pipeline, September 2018
Table 51: Editas Medicine Pipeline, September 2018.
Table 52: Editas Medicine SWOT
Table 53: Intellia Therapeutics Pipeline
Table 54: Intellia Therapeutics SWOT
Table 55: Homology Medicines Pipeline
Table 56: Homology Medicines SWOT
1.2 List of Figures
Figure 1: Nucleases Based on Protein-DNA Interactions
Figure 2: Transcription Activator-Like Effector Nucleases (TALENs)
Figure 3: The CRISPR/Cas System
Figure 4: CRISPR/Cas Binding Mechanism
Figure 5: Cas9 Orthologs
Figure 6: Gene Therapy Clinical Trials Worldwide by Vector
Figure 7: Multiple Sangamo Therapeutics Products Use an Albumin Targeting ‘Safe Harbor’ Approach
Figure 8: Gene Editing Landscape, Progression of Gene Therapy Applications
Figure 9: Gene Editing Landscape, Hemophilia A + B
Figure 10: SPK-9001: Factor IX Activity after SPK-9011 in 8 Participants that did not Show AAV Capsid-Directed Immune Response
Figure 11: Cost of Beta-Thalassemia Major Treatment as of NHS Tariffs at 2013/14 Prices
Figure 12: BCL11A is Involved in Fetal Hemoglobin Silencing

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