CONTENTS

6 EXECUTIVE SUMMARY

9 THE REGENERATIVE MEDICINE LANDSCAPE
9 Both the US and EU have specialized pathways for regenerative medicines
15 Modalities in regenerative medicine
18 Bibliography

22 KEY COMPANY PLAYERS IN REGENERATIVE MEDICINE
22 Across the marketed product portfolio, many players are emerging as leaders
29 Active developers in the pipeline include large and small companies
43 Bibliography

46 REGENERATIVE MEDICINE APPROVED PRODUCT TRENDS
46 Marketed products in regenerative medicine are dominated by devices
47 Bone grafts and cell therapies account for the largest concentration of marketed regenerative medicines
49 Musculoskeletal conditions lead the indications for approved or launched regenerative medicines
52 Availability of launched regenerative medicine products varies between therapies and devices
57 Bibliography

59 REGENERATIVE MEDICINE PIPELINE TRENDS
59 The pipeline is being driven by cell and gene therapies
59 Devices in development span multiple product types, but tend to focus on bone fractures and defects
61 Early-stage research is supporting regenerative medicine therapies
67 Noteworthy regenerative medicines are on the brink of approval
71 In vivo gene therapies represent nearly one-third of the regenerative medicine pharmaceuticals pipeline
72 The majority of regenerative medicines are in development for cancer or rare diseases
74 The regenerative medicine product type employed varies across therapy areas
75 Bibliography

78 DEAL-MAKING TRENDS IN REGENERATIVE MEDICINE
78 Regenerative medicine financing trends
85 Regenerative medicine mergers and acquisitions trends
91 Regenerative medicine alliances trends
98 Bibliography

100 APPENDIX
100 Scope
100 Methodology

LIST OF FIGURES
15 Figure 1: Multiple modalities in regenerative medicine
22 Figure 2: Active regenerative medicine device developers with approved or launched products
28 Figure 3: Active regenerative medicine therapy developers with approved or launched products
30 Figure 4: Active regenerative medicine device developers in the pipeline
33 Figure 5: Active regenerative medicine therapy developers in the pipeline, preclinical to pre-registration
43 Figure 6: Celprogen’s regenerative medicine pipeline by indication, May 2017
46 Figure 7: Devices comprise the majority of approved regenerative medicines
47 Figure 8: Global regenerative medicine approvals/launches, 2000–17*
48 Figure 9: Marketed regenerative medicine devices by product type
49 Figure 10: Marketed regenerative medicine therapies by product type
50 Figure 11: Leading indications among marketed regenerative medicine devices
51 Figure 12: Therapy area breakdown of marketed regenerative medicine therapies
52 Figure 13: Leading indications among marketed regenerative medicine therapies
53 Figure 14: The majority of regenerative medicine devices are available in North America
54 Figure 15: Approved/launched regenerative medicine devices by country/region
55 Figure 16: Asia-Pacific region leads in regenerative medicine therapy approvals/launches
56 Figure 17: Approved/launched regenerative medicine therapies globally
57 Figure 18: Approved/launched regenerative medicine therapies by country
59 Figure 19: Therapies comprise the majority of the regenerative medicine pipeline
60 Figure 20: Pipeline regenerative medicine devices by product type
61 Figure 21: Leading indications among pipeline regenerative medicine devices
62 Figure 22: Steady growth in the regenerative medicine therapy pipeline, 1995–2017
63 Figure 23: Proportion of the regenerative medicine pipeline by phase, 1995–2017
64 Figure 24: Phase status of ongoing clinical trials in regenerative medicine, May 2017
65 Figure 25: Regenerative medicine clinical trial activity by country, May 2017
66 Figure 26: Outcomes for completed regenerative medicine trials show most are successful, May 2017
67 Figure 27: Primary reasons for the termination of regenerative medicine clinical trials, May 2017
72 Figure 28: Pipeline regenerative medicine therapies by product type
73 Figure 29: Therapy area breakdown of pipeline regenerative medicine therapies, preclinical to pre-registration
74 Figure 30: Leading indications among pipeline regenerative medicine pharmaceuticals, preclinical to pre-registration
75 Figure 31: Treatment modalities vary across diseases in the regenerative medicine pipeline
79 Figure 32: Regenerative medicine financing activity by volume, 2012–16
80 Figure 33: Regenerative medicine financing activity by total deal value, 2012–16
85 Figure 34: Regenerative medicine M&A volume and value, 2012–16
86 Figure 35: Regenerative medicine M&A volume by modality, 2012–16
87 Figure 36: Biotech companies are the most active acquirers in regenerative medicine, 2012–16
91 Figure 37: Regenerative medicine partnerships by volume, 2012–16
92 Figure 38: Regenerative medicine partnerships by total deal value, 2012–16
97 Figure 39: Biotech-biotech collaborations dominate regenerative medicine partnerships, 2012–16
98 Figure 40: Active large company partners in regenerative medicine alliances, 2012–16

LIST OF TABLES
11 Table 1: US RMAT designations, June 2017
13 Table 2: EU regenerative medicine ATMP designations, June 2017
24 Table 3: Regenerative medicine device portfolios of the most active companies, May 2017
31 Table 4: Regenerative medicine device pipelines of the most active companies, May 2017
35 Table 5: Novartis’s regenerative medicine pipeline, May 2017
38 Table 6: Takeda’s regenerative medicine pipeline, May 2017
41 Table 7: Sanofi’s regenerative medicine pipeline, May 2017
69 Table 8: Regenerative medicines awaiting approval worldwide, May 2017
82 Table 9: Top venture financings in regenerative medicine, 2012–16
89 Table 10: Top 10 regenerative medicine M&As, 2012–16
94 Table 11: Regenerative medicine partnerships worth $1bn or higher, 2012–16