Rocket Pharmaceuticals
Rocket Pharmaceuticals sponsors 10 registered US clinical trials on ClinicalTrials.gov, 5 of them currently recruiting, and 2 completed. 0 of these trials are in Phase 3-4 (later-stage) and 7 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Danon Disease, with 2 trials.
Trial Pipeline
10 total, page 1 of 1
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
NCT07137338
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
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
NCT04525352
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
NCT04105166
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
NCT03812263
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 5 |
| Phase 2 | 2 |
What the Pipeline for Rocket Pharmaceuticals Shows
According to the ClinicalTrials.gov registry, Rocket Pharmaceuticals is linked to 10 US clinical trials across every stage of research activity. Of those, 5 studies are currently recruiting, about 50% of the sponsor's indexed portfolio, and 2 are already marked complete, representing roughly 20% of the total.
The phase mix for Rocket Pharmaceuticals reports 0 late-stage studies (Phase 3 and Phase 4 combined) and 7 earlier-phase studies (Phase 1 and Phase 2).
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.
How to read these pipeline numbers
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.
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.
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.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.