Sponsor pipeline

Spark Therapeutics, Inc. clinical trial pipeline

Data updated daily from ClinicalTrials.gov, last refresh 2026-10-16

Active trials by phase

Phase 1/2		2
Phase 3		1

Not charted: 1 active trial with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

NCT ID	Title	Condition	Status	Updated
Phase 3
NCT00999609	Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis	Inherited Retinal Dystrophy Due to RPE65 Mutations	Active, Not Recruiting	2025-04-23
Phase 1/2
NCT01208389	Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital…	Leber Congenital Amaurosis	Active, Not Recruiting	2025-04-29
NCT04093349	A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)	Pompe Disease	Active, Not Recruiting	2024-11-27
Not specified
NCT03432520	Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A	Hemophilia A	Active, Not Recruiting	2024-11-29

Get alerts when Spark Therapeutics, Inc.'s trials change

Status changes, date slips, and new filings: detected daily, delivered to your inbox.