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ROCKET PHARMACEUTICALS, INC. 8K

0001140361-26-030568

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Rocket Pharmaceuticals reported encouraging early safety results from the first three patients in its modified pivotal Phase 2 RP-A501 gene therapy trial for Danon disease and is working with the FDA on the path to trial completion.

Rocket Pharmaceuticals, Inc. announced a positive clinical safety update from the first three patients treated under a modified protocol in its global pivotal Phase 2 trial of RP-A501 for Danon disease, reporting that treatment at a recalibrated dose with a refined immunomodulatory regimen was well-tolerated with no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns observed, and stated that it is actively engaging with the FDA to determine the path for dosing additional patients and completing the trial, with a broader regulatory and Danon disease program update expected in the second half of 2026.

Filing Facts

CIK
1281895
Ticker
-
Form
8K
Source Type
sec
Accession
0001140361-26-030568
Alert Tier
6
ROCKET PHARMACEUTICALS, INC. 8K | ATTN