Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

A number of gene therapies have now received marketing authorizations in the US and Europe. The first to have received approval from the US and Europe was Glybera, which was approved in Europe in 2012 by the EMA for lipoprotein lipase (LPL) deficiency. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.

The drug uses a viral vector that inserts the wild-type lipoprotein lipase (LPL)-encoding gene in patients for whom the gene is mutated, causing the protein it encodes to be non-functional. Alternately, a sample of the patient’s cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein.

In subsequent years, more gene therapy products have been approved; this includes an oncolytic virus, a stem cell-based gene therapy, and, as of 2017, two chimeric antigen receptor (CAR)-T cell therapies. Researchers must overcome many technical challenges before gene therapy will be a practical approach to treating disease.

The current pipeline for gene therapies is very large, covering 985 active programs across all therapy areas. Although the majority (76%) are at either the Discovery or Preclinical stages and will not reach the market for a number of years, the general trend indicates that gene therapies will continue to grow in prominence. More approvals can be anticipated across various indications.

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 encoding LNGFR and HSV-TK) 36

3.15 Luxturna (Vortigern 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

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

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