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Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite Difficulties Faced by products such as Glybera and Strimvelis

Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite Difficulties Faced by products such as Glybera and Strimvelis

Summary

GBI Research’s latest report: “Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite Difficulties Faced by products such as Glybera and Strimvelis” discusses all gene therapies, including not only those which involve genetic modification, but also RNAi, CAR-T cell and aptamer-based therapies.

In 2017 alone, the marketing approvals of Kymriah, Yescarta and Luxturna added to this growing class of products. However, due to various challenges, progress in developing this technology and achieving commercial uptake over the past few decades has been slow.

In the short term, there is a shortfall in global gene and cell therapy manufacturing capacity, and in the long-term the personalized nature of many gene and cell therapies represents a further challenge that will not be met by the currently used, centralized biopharmaceutical manufacturing model.

Additionally, the very small number of patients in orphan genetic diseases that these therapies are especially well suited for has caused commercial difficulties for drugs such as Glybera and Strimvelis, and the high pricing of these therapies in response to the low patient number presented strong reimbursement difficulties.

However, the pipeline for gene therapies is robust; there are 985 in vivo gene therapies, and a further 354 CAR-T cell therapies currently in pipeline development. Most are at an early stage of development, with 76% at the Discovery or Preclinical stage.

There are also currently 23 gene therapy programs in Phase III development. This CBR report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.

Scope

  • Why do gene therapies still occupy only a minimal market share in their respective indications?
  • What can be learned from the gene therapies that have already reached the market, especially from products that have faced commercial difficulties, such as Glybera and Strimvelis?
  • What are the prospects of recent approvals such as Luxturna, Kymriah and Strimvelis?
  • What are the relative advantages and disadvantages of each vector type and which hold the most promise?
  • What proportion of the overall gene therapy R&D pipeline is occupied by each vector type?
  • How will the entry of gene therapies into the market change the global manufacturing landscape?
Reasons to buy
  • Understand the current status of the field of therapeutic gene therapies, and the relative clinical and commercial success of currently marketed products.
  • Assess the pipeline for gene therapies split by therapy area, vector type and intervention type, and stage of development. Additionally, a granular assessment of the pipeline is provided across the four major therapy areas for gene therapy: oncology, central nervous system disorders, ophthalmology, and genetic disorders.
  • Gain a picture of the current competitive landscape, with a detailed breakdown of companies actively involved in the gene therapy pipeline.
  • Understand the level of involvement in the landscape on the part of big pharma companies, and the extent to how gene therapies fit into the overall portfolios of companies in this field.
  • Understand the strategic consolidations landscape in gene therapies across the past decade"


1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Gene Therapy Overview
2.1 Types of Gene Therapy
2.1.1 Types of Intervention
2.1.2 Types of Vector
3 Currently Approved Gene Therapies
3.1 Glybera (alipogene tiparvovec)
3.2 Kynamro (mipomersen)
3.3 Macugen (pegaptanib)
3.4 Vitravene (fomivirsen)
3.5 Gendicine (rAd-p53)
3.6 Oncorine (rAd5-H101)
3.7 Neovasculgen (Pl-VEGF165)
3.8 Exondys 51 (eteplirsen)
3.9 Spinraza (nusinersen)
3.10 Strimvelis (GSK-2696273)
3.11 Kymriah (tisagenlecleucel)
3.12 Yescarta (axicabtagene ciloleucel)
3.13 Imlygic (talimogene laherparepvec)
3.14 Zalmoxis (Allogenic T cells encodng LNGFR and HSV-TK)
3.15 Luxturna (voretigene neparvovec)
4 Gene Therapy Production Strategies
4.1 Production of Viral Vectors
4.1.1 Case Study: Challenges in the Manufacture of AAV Vectors
4.2 Production of Cell-based Gene Therapies
5 Challenges to Gene Therapy Development
6 Gene Therapy Pipeline and Emerging Technologies
6.1 Gene Therapy Pipeline by Therapy Area and Stage of Development
6.2 Gene Therapy Pipeline by Intervention and Vector Type
6.3 Pipeline for CAR-T Cell Therapies
6.4 Company Positioning
6.4.1 Companies by Therapy Area
6.4.2 Companies by Stage of Development
6.4.3 Companies by Intervention Type
6.4.4 Companies by Vector Type
6.4.5 Companies Developing CAR-T Cell Therapies by Stage
6.5 Early Genome Editing Technologies
6.5.1 Zinc Finger Nucleases
6.5.2 TALEN
6.5.3 CRISPR-Cas9
7 Strategic Consolidations
7.1 Licensing Deals
7.1.1 Licensing Deals by Region Value and Year
7.1.2 Licensing Deals by Stage of Development and Value
7.1.3 Licensing Deals by Intervention and Vector Type
7.2 Co-development deals
7.2.1 Co-development Deals by Region, Year and Value
7.2.2 Co-development Deals by Stage of Development and Value
7.2.3 Co-development Deals by Intervention and Vector Type
8 Conclusion
9 Appendix
9.1 References
9.2 About GBI Research
List of Tables
1.1 List of Tables
Table 1: Gene Therapies, Global, Methods of Gene Silencing, 2017
Table 2: Gene Therapies, Global, Types of Viral Vector for Gene Therapy Delivery, 2017
Table 3: Gene Therapies, Global, Types of Non-Viral Vector for Gene Therapy Delivery, 2017
Table 4: Gene Therapies, Global, Clinical Characteristics of Glybera, 2017
Table 5: Gene Therapies, Global, Clinical Characteristics of Kynamro, 2017
Table 6: Gene Therapies, Global, Clinical Characteristics of Macugen, 2017
Table 7: Gene Therapies, Global, Clinical Characteristics of Vitravene, 2017
Table 8: Gene Therapies, Global, Clinical Characteristics of Gendicine, 2017
Table 9: Gene Therapies, Global, Clinical Characteristics of Oncorine, 2017
Table 10: Gene Therapies, Global, Clinical Characteristics of Neovasculgen, 2017
Table 11: Gene Therapies, Global, Clinical Characteristics of Exondys 51, 2017
Table 12: Gene Therapies, Global, Clinical Characteristics of Spinraza, 2017
Table 13: Gene Therapies, Global, Clinical Characteristics of Strimvelis, 2017
Table 14: Gene Therapies, Global, Clinical Characteristics of Kymriah, 2017
Table 15: Gene Therapies, Global, Clinical Characteristics of Yescarta, 2017
Table 16: Gene Therapies, Global, Clinical Characteristics of Imlygic, 2017
Table 17: Gene Therapies, Global, Clinical Characteristics of Zalmoxis, 2017
Table 18: Gene Therapies, Global, Clinical Characteristics of Luxturna, 2017
Table 19: Gene Therapies, Global, Impurities in the AAV Production Process, 2017
Table 20: Gene Therapies, Global, Overall Challenges to Gene Therapy Development, 2017
Table 21: Gene Therapies, Global, Comparison of CRISPR-Cas9, ZFNs and TALENs as Gene Editing Platforms, 2017
List of Figures
1.2 List of Figures
Figure 1: Gene Therapies, Global, Definition of Gene Therapy, 2017
Figure 2: Gene Therapies, Global, Clinical Trials Approved for Gene Therapies, 1989-2012
Figure 3: Gene Therapies, Global, Timeline of Key Events in Gene Therapy Research, 1944-2017
Figure 4: Gene Therapies, Global, Types of Gene Therapy-based Intervention, 2017
Figure 5: Gene Therapies, Global, Types of Vector for Gene Therapy Delivery, 2017
Figure 6: Gene Therapies, Global, Specific Types of Vector for Gene Therapy Delivery, 2017
Figure 7: Gene Therapies, Global, Production of Viral Vectors for Gene Therapy; Genome Modification, 2017
Figure 8: Gene Therapies, Global, Viral Gene Therapy Vector Manufacture; General Production and Purification Scheme, 2017
Figure 9: Gene Therapies, Global, Gene Therapy Pipeline by Therapy Area and Stage of Development, 2017
Figure 10: Gene Therapies, Global, Gene Therapy Pipeline by Therapy Area and Stage of Development, 2017
Figure 11: Gene Therapies, Global, Gene Therapy Pipeline by Vector and Intervention Type, 2017
Figure 12: Gene Therapies, Global, Gene Therapy Pipeline by Vector and Therapy Area, 2017
Figure 13: Gene Therapies, Global, Gene Therapy Pipeline by Intervention Type and Therapy Area, 2017
Figure 14: Gene Therapies, Global, CAR-T Cell Therapies by Stage of development, 2017
Figure 15: Gene Therapies, Global, CAR-T Cell Therapies by Molecular Target, 2017
Figure 16: Gene Therapies, Global, Gene Therapy Pipeline Companies by Type and Level of Diversification, 2017
Figure 17: Gene Therapies, Global, Gene Therapy Pipeline by Company and Therapy Area, 2017
Figure 18: Gene Therapies, Global, Gene Therapy Pipeline by Company and Stage of Development, 2017
Figure 19: Gene Therapies, Global, Gene Therapy Pipeline by Company and Intervention Type, 2017
Figure 20: Gene Therapies, Global, Gene Therapy Pipeline by Company and Vector Type, 2017
Figure 21: Gene Therapies, Global, CAR-T Cell Therapy Pipeline by Company and Stage of development, 2017
Figure 22: Gene Therapies, Global, Licensing Deals by Year, 2006-2017
Figure 23: Gene Therapies, Global, Licensing Deals by Stage of Development and Value, 2006-2017
Figure 24: Gene Therapies, Global, Licensing Deals by Vector type, 2006-2017
Figure 25: Gene Therapies, Global, Licensing Deals by Intervention type, 2006-2017
Figure 26: Gene Therapies, Global, Co-Development Deals by Year, 2006-2017
Figure 27: Gene Therapies, Global,Co-Development Deals by Year and Value, 2006-2017
Figure 28: Gene Therapies, Global,Co-Development Deals by Vector, 2006-2017
Figure 29: Gene Therapies, Global,Co-Development Deals by Vector, 2006-2017

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