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

Home/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

Treatments, Research & Development (Pharma & Healthcare), Pharmaceutical Manufacturing, Pharmacy, Pharma & Healthcare

Publication ID:

CBR0118001

Publication Date:

January 01, 2018

Pages:

Publisher:

CBR Pharma Insights

Region:

Global

$2,995.00

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Description

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

Table of Contents

1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3
2 Gene Therapy Overview 5
2.1 Types of Gene Therapy 7
2.1.1 Types of Intervention 8
2.1.2 Types of Vector 13
3 Currently Approved Gene Therapies 22
3.1 Glybera (alipogene tiparvovec) 22
3.2 Kynamro (mipomersen) 23
3.3 Macugen (pegaptanib) 24
3.4 Vitravene (fomivirsen) 25
3.5 Gendicine (rAd-p53) 26
3.6 Oncorine (rAd5-H101) 26
3.7 Neovasculgen (Pl-VEGF165) 27
3.8 Exondys 51 (eteplirsen) 28
3.9 Spinraza (nusinersen) 29
3.10 Strimvelis (GSK-2696273) 30
3.11 Kymriah (tisagenlecleucel) 32
3.12 Yescarta (axicabtagene ciloleucel) 33
3.13 Imlygic (talimogene laherparepvec) 34
3.14 Zalmoxis (Allogenic T cells encodng LNGFR and HSV-TK) 36
3.15 Luxturna (voretigene neparvovec) 37
4 Gene Therapy Production Strategies 38
4.1 Production of Viral Vectors 38
4.1.1 Case Study: Challenges in the Manufacture of AAV Vectors 40
4.2 Production of Cell-based Gene Therapies 42
5 Challenges to Gene Therapy Development 44
6 Gene Therapy Pipeline and Emerging Technologies 46
6.1 Gene Therapy Pipeline by Therapy Area and Stage of Development 46
6.2 Gene Therapy Pipeline by Intervention and Vector Type 48
6.3 Pipeline for CAR-T Cell Therapies 51
6.4 Company Positioning 52
6.4.1 Companies by Therapy Area 53
6.4.2 Companies by Stage of Development 54
6.4.3 Companies by Intervention Type 54
6.4.4 Companies by Vector Type 56
6.4.5 Companies Developing CAR-T Cell Therapies by Stage 57
6.5 Early Genome Editing Technologies 57
6.5.1 Zinc Finger Nucleases 58
6.5.2 TALEN 58
6.5.3 CRISPR-Cas9 59
7 Strategic Consolidations 62
7.1 Licensing Deals 62
7.1.1 Licensing Deals by Region Value and Year 62
7.1.2 Licensing Deals by Stage of Development and Value 64
7.1.3 Licensing Deals by Intervention and Vector Type 65
7.2 Co-development deals 66
7.2.1 Co-development Deals by Region, Year and Value 66
7.2.2 Co-development Deals by Stage of Development and Value 68
7.2.3 Co-development Deals by Intervention and Vector Type 69
8 Conclusion 71
9 Appendix 72
9.1 References 72
9.2 About GBI Research 79

List of Tables
1.1 List of Tables
Table 1: Gene Therapies, Global, Methods of Gene Silencing, 2017 10
Table 2: Gene Therapies, Global, Types of Viral Vector for Gene Therapy Delivery, 2017 15
Table 3: Gene Therapies, Global, Types of Non-Viral Vector for Gene Therapy Delivery, 2017 19
Table 4: Gene Therapies, Global, Clinical Characteristics of Glybera, 2017 23
Table 5: Gene Therapies, Global, Clinical Characteristics of Kynamro, 2017 24
Table 6: Gene Therapies, Global, Clinical Characteristics of Macugen, 2017 25
Table 7: Gene Therapies, Global, Clinical Characteristics of Vitravene, 2017 25
Table 8: Gene Therapies, Global, Clinical Characteristics of Gendicine, 2017 26
Table 9: Gene Therapies, Global, Clinical Characteristics of Oncorine, 2017 27
Table 10: Gene Therapies, Global, Clinical Characteristics of Neovasculgen, 2017 27
Table 11: Gene Therapies, Global, Clinical Characteristics of Exondys 51, 2017 29
Table 12: Gene Therapies, Global, Clinical Characteristics of Spinraza, 2017 30
Table 13: Gene Therapies, Global, Clinical Characteristics of Strimvelis, 2017 32
Table 14: Gene Therapies, Global, Clinical Characteristics of Kymriah, 2017 33
Table 15: Gene Therapies, Global, Clinical Characteristics of Yescarta, 2017 34
Table 16: Gene Therapies, Global, Clinical Characteristics of Imlygic, 2017 35
Table 17: Gene Therapies, Global, Clinical Characteristics of Zalmoxis, 2017 36
Table 18: Gene Therapies, Global, Clinical Characteristics of Luxturna, 2017 37
Table 19: Gene Therapies, Global, Impurities in the AAV Production Process, 2017 41
Table 20: Gene Therapies, Global, Overall Challenges to Gene Therapy Development, 2017 44
Table 21: Gene Therapies, Global, Comparison of CRISPR-Cas9, ZFNs and TALENs as Gene Editing Platforms, 2017 57

List of Figures
1.2 List of Figures
Figure 1: Gene Therapies, Global, Definition of Gene Therapy, 2017 5
Figure 2: Gene Therapies, Global, Clinical Trials Approved for Gene Therapies, 1989-2012 6
Figure 3: Gene Therapies, Global, Timeline of Key Events in Gene Therapy Research, 1944-2017 7
Figure 4: Gene Therapies, Global, Types of Gene Therapy-based Intervention, 2017 8
Figure 5: Gene Therapies, Global, Types of Vector for Gene Therapy Delivery, 2017 14
Figure 6: Gene Therapies, Global, Specific Types of Vector for Gene Therapy Delivery, 2017 14
Figure 7: Gene Therapies, Global, Production of Viral Vectors for Gene Therapy; Genome Modification, 2017 39
Figure 8: Gene Therapies, Global, Viral Gene Therapy Vector Manufacture; General Production and Purification Scheme, 2017 40
Figure 9: Gene Therapies, Global, Gene Therapy Pipeline by Therapy Area and Stage of Development, 2017 46
Figure 10: Gene Therapies, Global, Gene Therapy Pipeline by Therapy Area and Stage of Development, 2017 47
Figure 11: Gene Therapies, Global, Gene Therapy Pipeline by Vector and Intervention Type, 2017 48
Figure 12: Gene Therapies, Global, Gene Therapy Pipeline by Vector and Therapy Area, 2017 49
Figure 13: Gene Therapies, Global, Gene Therapy Pipeline by Intervention Type and Therapy Area, 2017 50
Figure 14: Gene Therapies, Global, CAR-T Cell Therapies by Stage of development, 2017 51
Figure 15: Gene Therapies, Global, CAR-T Cell Therapies by Molecular Target, 2017 51
Figure 16: Gene Therapies, Global, Gene Therapy Pipeline Companies by Type and Level of Diversification, 2017 52
Figure 17: Gene Therapies, Global, Gene Therapy Pipeline by Company and Therapy Area, 2017 53
Figure 18: Gene Therapies, Global, Gene Therapy Pipeline by Company and Stage of Development, 2017 54
Figure 19: Gene Therapies, Global, Gene Therapy Pipeline by Company and Intervention Type, 2017 55
Figure 20: Gene Therapies, Global, Gene Therapy Pipeline by Company and Vector Type, 2017 56
Figure 21: Gene Therapies, Global, CAR-T Cell Therapy Pipeline by Company and Stage of development, 2017 57
Figure 22: Gene Therapies, Global, Licensing Deals by Year, 2006-2017 63
Figure 23: Gene Therapies, Global, Licensing Deals by Stage of Development and Value, 2006-2017 64
Figure 24: Gene Therapies, Global, Licensing Deals by Vector type, 2006-2017 65
Figure 25: Gene Therapies, Global, Licensing Deals by Intervention type, 2006-2017 66
Figure 26: Gene Therapies, Global, Co-Development Deals by Year, 2006-2017 67
Figure 27: Gene Therapies, Global,Co-Development Deals by Year and Value, 2006-2017 68
Figure 28: Gene Therapies, Global,Co-Development Deals by Vector, 2006-2017 69
Figure 29: Gene Therapies, Global,Co-Development Deals by Vector, 2006-2017 70

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