Spark Therapeutics
Trial Pipeline
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT04093349
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
NCT03432520
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
NCT00999609
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
NCT01208389
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
NCT03734588
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
NCT02341807
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 3 | 1 |
Therapeutic Areas
What the Pipeline for Spark Therapeutics Shows
According to the ClinicalTrials.gov registry, Spark Therapeutics is linked to 6 US clinical trials across every stage of research activity. Of those, 0 studies are currently recruiting — about 0% of the sponsor's indexed portfolio — and 2 are already marked complete, representing roughly 33% of the total. Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
The phase mix for Spark Therapeutics reports 1 late-stage studies (Phase 3 and Phase 4 combined) and 4 earlier-phase studies (Phase 1 and Phase 2). A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work — safety, dosing, and early signal detection — and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
The top therapeutic focus area indexed for Spark Therapeutics is Leber Congenital Amaurosis with 2 linked trials, and 9 other condition areas appear in the top list above. That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice — verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology — how this data is sourced, computed, and verified.