Rocket Pharmaceuticals
Trial Pipeline
PKP2-ACM Natural History Study
NCT06644742
Danon Disease Natural History Study
NCT06214507
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
NCT06092034
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
NCT05885412
Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
NCT06282432
Gene Therapy for Fanconi Anemia, Complementation Group A
NCT04248439
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 2 |
What the Pipeline for Rocket Pharmaceuticals Shows
According to the ClinicalTrials.gov registry, Rocket Pharmaceuticals is linked to 6 US clinical trials across every stage of research activity. Of those, 4 studies are currently recruiting — about 67% of the sponsor's indexed portfolio — and 0 are already marked complete, representing roughly 0% 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 Rocket Pharmaceuticals reports 0 late-stage studies (Phase 3 and Phase 4 combined) and 3 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 Rocket Pharmaceuticals is Danon Disease with 2 linked trials, and 7 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.