---
schema_version: "secwatch.filing_event.v1"
accession: "0001140361-25-032012"
form_type: "8-K"
ticker: "RCKT"
cik: "0001281895"
company_name: "ROCKET PHARMACEUTICALS, INC."
filed_at: "2025-08-20T23:59:59+00:00"
generated_at: "2026-05-17T11:06:17.000342+00:00"
event_type: "regulatory"
sentiment: "positive"
materiality_score: 0.85
calibrated_materiality_score: 0.85
confidence: "high"
source: SEC EDGAR
---

# Rocket Pharmaceuticals: FDA lifts clinical hold on Phase 2 trial of RP-A501 for Danon disease

## Summary
- FDA lifted clinical hold in under 3 months; trial resumes with recalibrated dose 3.8x10^13 GC/kg for 3 patients with 4-week intervals.
- Revised immunomodulatory regimen: discontinue prophylactic C3 inhibitor, maintain sirolimus/rituximab/steroids, lower eculizumab threshold.
- Phase 2 trial is 12-patient single-arm; co-primary endpoint: LAMP2 expression and left ventricular mass reduction; key secondary: troponin change.
- Six patients previously treated at 6.7x10^13 GC/kg; three additional at lower dose to be treated sequentially.
- Natural history study running concurrently; all patients require 3-month pre-treatment run-in for biomarker assessment.

## SEC filing metadata
- accession: 0001140361-25-032012
- form_type: 8-K
- ticker: RCKT
- cik: 0001281895
- company_name: ROCKET PHARMACEUTICALS, INC.
- filed_at: 2025-08-20T23:59:59+00:00
- event_type: regulatory
- sentiment: positive
- materiality_score: 0.85
- calibrated_materiality_score: 0.85
- confidence: high
- sec_items: 8.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1281895/000114036125032012/0001140361-25-032012-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1281895/000114036125032012/ef20054253_8k.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001140361-25-032012
- JSON: https://secwatch.observer/filing/0001140361-25-032012.json
- Plain text: https://secwatch.observer/filing/0001140361-25-032012.txt

This AI-assisted summary is a reading aid. Review the linked SEC EDGAR filing before relying on any specific claim.