Table of Contents
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
3.1 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