Spark Therapeutics, Inc.
15
0
5
8
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Moderate Risk
Score: 50/100
13.3%
2 terminated/withdrawn out of 15 trials
80.0%
-6.5% vs industry average
13%
2 trials in Phase 3/4
50%
4 of 8 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (15)
A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Role: lead
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Role: lead
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Role: lead
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Role: lead
A Gene Transfer Study for Hemophilia A
Role: lead
Study of a Gene Therapy Treatment for Hemophilia A
Role: lead
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Role: lead
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Role: lead
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Role: lead
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
Role: lead
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Role: lead
Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Role: lead
Safety Study in Subjects With Leber Congenital Amaurosis
Role: lead
LTFU for Gene Transfer Subjects With Hemophilia B
Role: lead
Hemophilia B Gene Therapy With AAV8 Vector
Role: lead
All 15 trials loaded