ROCKET PHARMACEUTICALS, INC.

Highlights Recent Progress

Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026

KRESLADI™ for severe LAD-I on track for March 28, 2026 PDUFA date

Dosing of first patient in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026

Cash, cash equivalents and investments of approximately $188.9M;

expected operational runway into the second quarter of 2027

CRANBURY, NJ–February 26, 2026–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the fourth quarter and year ended December 31, 2025.

“In 2025, we strengthened Rocket’s leadership in cardiovascular gene therapy, supported by more than five years of clinical experience in Danon disease and continued advancement across our PKP2-ACM and BAG3-DCM programs,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “Our focus remains on rigorous clinical execution across three cardiovascular programs, including resuming the Phase 2 pivotal clinical trial in Danon disease, initiating the Phase 1 clinical trial in BAG3-DCM, and advancing alignment with the FDA on the pivotal study design for PKP2-ACM. As we move through 2026, Rocket is well positioned to advance three high-impact cardiovascular programs and thoughtfully expand our cardiac gene therapy pipeline.”