Table of Contents
Table of Contents 3
List of Tables 6
List of Figures 6
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
Spark Therapeutics Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 8
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 10
Spark Therapeutics Inc, Medical Devices Deals, 2012 to YTD 2018 11
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 12
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deal Details 14
Venture Financing 14
Spark Therapeutics Raises USD73 Million in Series B Financing 14
Spark Therapeutics Raises US$50 Million In Venture Financing 16
Partnerships 17
Spark Therapeutics Enters into partnership with University of Massachusetts Medical School 17
Licensing Agreements 18
Novartis Enters into Licensing and Supply Agreement with Spark Therapeutics 18
Spark Therapeutics Enters into Licensing Agreement with Genethon 19
Spark Therapeutics Enters into Licensing Agreement with Selecta Biosciences 20
Pfizer Amends Licensing Agreement with Spark Therapeutics 22
Spark Therapeutics Enters into Licensing Agreement with University of Pennsylvania for Proviral Plasmids 24
Spark Therapeutics Enters Into Licensing Agreement With Genable Technologies 25
Spark Therapeutics Amends Licensing Agreement with University of Iowa Research Foundation 26
Spark Therapeutics Enters into Licensing Agreement with Children’s Hospital of Philadelphia 28
Spark Therapeutics Amends Licensing Agreement with University of Pennsylvania 29
Benitec Biopharma Enters Into Licensing Agreement With Genable Technologies 30
Equity Offering 31
Spark Therapeutics Raises USD402.5 Million in Public Offering of Shares 31
Selecta Biosciences to Raise USD5 Million in Private Placement of Shares 33
Spark Therapeutics Raises USD181 Million in Public Offering of Shares 35
Spark Therapeutics Raises USD107 Million in Public Offering of Shares 37
Genable Tech to Raise USD15.7 Million in Financing 39
Spark Therapeutics Raises USD185 Million in IPO 40
Asset Transactions 42
JAZZ Pharma to Acquire Rare Pediatric Disease Priority Review Voucher from Spark Therapeutics for USD110 Million 42
Acquisition 43
Spark Therapeutics Acquires Genable Technologies for USD15.3 Million 43
Spark Therapeutics Inc – Key Competitors 44
Spark Therapeutics Inc – Key Employees 45
Spark Therapeutics Inc – Locations And Subsidiaries 46
Head Office 46
Other Locations & Subsidiaries 46
Recent Developments 47
Strategy And Business Planning 47
Oct 11, 2018: Governor Wolf Announces Spark Therapeutics to Expand in West Philadelphia with New Facility, 500 New Jobs 47
Financial Announcements 48
Aug 07, 2018: Spark Therapeutics Reports Second Quarter 2018 Financial Results and Recent Business Progress 48
May 08, 2018: Spark Therapeutics Reports First Quarter 2018 Financial Results and Recent Business Progress 51
Feb 20, 2018: Spark Therapeutics Reports 2017 Financial Results and Recent Business Progress 53
Nov 07, 2017: Spark Therapeutics Reports Third Quarter 2017 Financial Results and Recent Business Progress 55
Aug 02, 2017: Spark Therapeutics Reports Second Quarter 2017 Financial Results and Recent Business Progress 57
May 09, 2017: Spark Therapeutics Reports First Quarter 2017 Financial Results and Recent Business Progress 60
Feb 22, 2017: Spark Therapeutics Reports 2016 Financial Results and Business Highlights 62
Corporate Communications 64
Jan 04, 2018: Spark Therapeutics Announces Addition of Robert J. Perez to Board of Directors 64
Sep 25, 2017: Spark Therapeutics Further Expands Gene Therapy Expertise with Appointment of Federico Mingozzi, Ph.D., as Chief Scientific Officer 65
Product News 66
12/19/2017: FDA Approves Spark Therapeutics LUXTURNA (voretigene neparvovec-rzyl), a One-time Gene Therapy for Patients with Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy 66
11/02/2017: Spark Therapeutics to Participate in Multiple Upcoming Conferences 68
10/12/2017: FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease 69
07/10/2017: Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent and Sustained Response at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress 71
06/23/2017: Spark Therapeutics to Present Updated Hemophilia B Data at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress 73
04/06/2017: Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial Suggesting Consistent and Sustained Levels of Factor IX Activity at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium 74
04/04/2017: Spark Therapeutics to Present Updated Hemophilia B Data at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium 75
03/26/2018: Boy with Inherited Blindness First to Receive FDA-Approved Gene Therapy at Bascom Palmer 76
03/21/2018: Childrens Hospital Los Angeles Treats First Patient on the West Coast Using Gene Therapy 77
03/20/2018: Mass. Eye and Ear performs first FDA-approved gene therapy procedure for inherited disease 78
03/05/2018: Horizon Blue Cross Blue Shield Provides Update on Voretigene neparvovec-rzyl (Luxturna) 79
01/03/2018: Spark Therapeutics Announces First-of-their-kind Programs to Improve Patient Access to LUXTURNA (voretigene neparvovec-rzyl), a One-time Gene Therapy Treatment 80
01/03/2018: Spark’s Gene Therapy Treatment for Blindness LUXTURNA to Cost $850,000 83
Product Approvals 85
Oct 19, 2017: Spark Therapeutics: Gene therapy continues to advance 85
Jul 31, 2017: Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA (voretigene neparvovec) 86
Jul 20, 2017: Spark Therapeutics Receives Rare Pediatric Disease Designation for Investigational LUXTURNA (voretigene neparvovec) from FDA 88
Jul 17, 2017: Spark Therapeutics’ Biologics License Application for Investigational Voretigene Neparvovec Accepted for Filing by FDA 89
May 18, 2017: Spark Therapeutics Completes Rolling Biologics License Application Submission to FDA for Investigational Gene Therapy Voretigene 91
Mar 01, 2017: Spark Therapeutics and Pfizer Announce that SPK-9001, an Investigational Hemophilia B Medicine, has been Granted Access to the PRIority MEdicines (PRIME) Program by the European Medicines Agency 92
Clinical Trials 93
Oct 08, 2018: Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate 93
Feb 23, 2018: Spark Therapeutics to Participate in Multiple Conferences in March 94
Nov 10, 2017: Three-year Follow-up Phase 3 Data Provide Additional Information on Efficacy, Durability and Safety of Investigational LUXTURNA (voretigene neparvovec) in Patients with Biallelic RPE65-mediated Inherited Retinal Disease 95
Oct 12, 2017: Spark Therapeutics (ONCE) Trading of Stock Halted as FDA Advisory Committee Reviews Investigational Gene Therapy 97
Sep 06, 2017: Spark Therapeutics Announces Publication of Study Confirming Novel Tests Validity, Reliability and Ability to Detect Change in Functional Vision 99
Jul 13, 2017: Spark Therapeutics Announces Publication in The Lancet of Pivotal Phase 3 Clinical Trial Data for Investigational Voretigene Neparvovec 101
Jan 09, 2017: Spark Therapeutics Announces U.S. Orphan Drug Designation Amendment and Study Updates for Lead Investigational Gene Therapy 103
Appendix 105
Methodology 105
About GlobalData 105
Contact Us 105
Disclaimer 105
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Key Facts, 2017 2
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
Spark Therapeutics Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 8
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
Spark Therapeutics Inc, Deals By Therapy Area, 2012 to YTD 2018 10
Spark Therapeutics Inc, Medical Devices Deals, 2012 to YTD 2018 11
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 12
Spark Therapeutics Raises USD73 Million in Series B Financing 14
Spark Therapeutics Raises US$50 Million In Venture Financing 16
Spark Therapeutics Enters into partnership with University of Massachusetts Medical School 17
Novartis Enters into Licensing and Supply Agreement with Spark Therapeutics 18
Spark Therapeutics Enters into Licensing Agreement with Genethon 19
Spark Therapeutics Enters into Licensing Agreement with Selecta Biosciences 20
Pfizer Amends Licensing Agreement with Spark Therapeutics 22
Spark Therapeutics Enters into Licensing Agreement with University of Pennsylvania for Proviral Plasmids 24
Spark Therapeutics Enters Into Licensing Agreement With Genable Technologies 25
Spark Therapeutics Amends Licensing Agreement with University of Iowa Research Foundation 26
Spark Therapeutics Enters into Licensing Agreement with Children’s Hospital of Philadelphia 28
Spark Therapeutics Amends Licensing Agreement with University of Pennsylvania 29
Benitec Biopharma Enters Into Licensing Agreement With Genable Technologies 30
Spark Therapeutics Raises USD402.5 Million in Public Offering of Shares 31
Selecta Biosciences to Raise USD5 Million in Private Placement of Shares 33
Spark Therapeutics Raises USD181 Million in Public Offering of Shares 35
Spark Therapeutics Raises USD107 Million in Public Offering of Shares 37
Genable Tech to Raise USD15.7 Million in Financing 39
Spark Therapeutics Raises USD185 Million in IPO 40
JAZZ Pharma to Acquire Rare Pediatric Disease Priority Review Voucher from Spark Therapeutics for USD110 Million 42
Spark Therapeutics Acquires Genable Technologies for USD15.3 Million 43
Spark Therapeutics Inc, Key Competitors 44
Spark Therapeutics Inc, Key Employees 45
Spark Therapeutics Inc, Subsidiaries 46
List of Figures
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 2
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 2
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 2
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 2
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 8
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
Spark Therapeutics Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 10
Spark Therapeutics Inc, Medical Devices Deals, 2012 to YTD 2018 11