CONTENTS

6 EXECUTIVE SUMMARY

8 GENE THERAPY DEAL-MAKING ACROSS THE INDUSTRY
8 Deal volume is generally increasing, supported by alliances
9 Outlier transactions, especially in acquisitions, have contributed to peaks in gene therapy values in certain years

12 GENE THERAPY ACQUISITIONS
12 There has been a steady increase in the volume of gene therapy acquisitions
12 Acquisitions have reached multibillion-dollar values, thanks to outlier transactions
17 Both gene therapies, and cell and gene therapies, are the subjects of acquisitions
19 Besides oncology, another active therapy area target in acquisitions is blood diseases
20 Bibliography

22 GENE THERAPY ALLIANCES
22 Aggregate value for alliances has reached $30bn
23 As time goes on, upfront payments represent a lower proportion of total deal value
25 The top 10 deals captured a large part of the total value
29 Over time, cell and gene therapies have become more of a focus in partnerships
30 Oncology gene therapies are subject of the most alliances
34 Bellicum, a CAR-T developer, is the most active gene therapy in-licenser
36 Bibliography

37 GENE THERAPY FINANCINGS
37 Companies working in gene therapy have raised $14bn through various financing types
40 Gene therapy developers were more active in financing
43 Cell and gene therapy companies lead the top 10 grossing IPOs
48 Gene editing was well represented in top 10 gene therapy venture rounds
52 Bibliography

53 DEAL-MAKING CASE STUDIES IN GENE THERAPY
53 Pfizer commits to gene therapy with Bamboo Therapeutics
54 CAR-T is worth billions to Gilead
56 Biogen and the University of Pennsylvania form a lucrative industry/academic partnership
58 Vertex turns to gene editing as another way to extend its cystic fibrosis franchise
59 Bibliography

61 APPENDIX
61 Scope
61 Methodology

LIST OF FIGURES
8 Figure 1: Gene therapy deals by volume and deal type, 2012–17
9 Figure 2: Most gene therapy deal activity is through partnerships: proportion of gene therapy deal volume by deal type, 2012–17*
10 Figure 3: Gene therapy deals by value and deal type, 2012–17
11 Figure 4: Acquisitions in gene therapy represent the largest value: proportion of gene therapy deal value by deal type, 2012–17
12 Figure 5: Gene therapy acquisition volume and value, 2012–17*
18 Figure 6: Gene therapy acquisition volume by category, 2012–17*
19 Figure 7: Gene therapy acquisition volume proportion by gene therapy category, 2012–17*
20 Figure 8: Gene therapy acquisition volume by therapy area, 2012–17*
23 Figure 9: Gene therapy alliances volume and value, 2012–17*
24 Figure 10: Upfront payments are decreasing as a proportion of total value in gene therapy deals, 2012–17*
25 Figure 11: Upfront and total value averages on gene therapy deals, 2012–17*
29 Figure 12: Gene therapy alliances volume by category, 2012–17*
30 Figure 13: Cell and gene therapies are taking a greater share of alliances, 2012–17*
31 Figure 14: Gene therapy alliances volume by therapy area, 2012–17*
32 Figure 15: Annual gene therapy alliances volume by therapy area, 2012–17*
33 Figure 16: Gene therapy deals by therapy area and category, 2012–17*
34 Figure 17: Cell and gene therapies in oncology: alliances volume and value, 2012–17*
35 Figure 18: Leading companies in gene therapy in-licensing, 2012–17*
36 Figure 19: Big Pharma and Mid Pharma gene therapy in-licensing volume by category, 2012–17*
37 Figure 20: Gene therapy financings by volume, 2012–17*
38 Figure 21: Gene therapy financings by value, 2012–17*
39 Figure 22: Public offerings represent a smaller share in volume, but dominate the value in gene therapy, 2012–17*
40 Figure 23: Gene therapy average per financing values, 2012–17*
41 Figure 24: Gene therapy financing volume by category, 2012–17*
42 Figure 25: Gene therapy financing volume proportion by gene therapy category, 2012–17*
43 Figure 26: Public offering volume proportion by gene therapy category, 2012–17*
43 Figure 27: Private placement volume proportion by gene therapy category, 2012–17*
53 Figure 28: Pfizer and Bamboo Therapeutics: deal structure
55 Figure 29: Gilead and Kite Pharma: deal structure
57 Figure 30: Biogen and the University of Pennsylvania: deal structure
58 Figure 31: Vertex and CRISPR Therapeutics: deal structure

LIST OF TABLES
15 Table 1: Top 10 gene therapy acquisitions by value, 2012–17*
27 Table 2: Top 10 gene therapy alliances by value, 2012–17*
45 Table 3: Top 10 gene therapy IPOs, 2012–17*
49 Table 4: Top 10 gene therapy venture financings, 2012–17*