ROCKET PHARMACEUTICALS, INC.

Highlights Recent Progress

All three initial patients treated with RP-A501 under the modified pivotal Phase 2 protocol completed the protocol-defined safety observation period; no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns observed

Rocket is actively engaging with the FDA to align on the path to rapidly completing the pivotal Phase 2 trial in Danon disease

Multiple clinical and regulatory milestones remain on track for the second half of 2026, including a broader Danon disease program update, a regulatory update for the planned RP-A601 pivotal study in PKP2-ACM and initial patient dosing with RP-A701 in BAG3-DCM

FDA approval of KRESLADI™ and subsequent monetization of the Priority Review Voucher generated $180 million in non-dilutive capital; focused preparations are underway for commercial availability and patient onboarding beginning in the fourth quarter of 2026

Cash, cash equivalents and investments of approximately $283.7 million; expected cash runway into the second quarter of 2028

CRANBURY, NJ–August 10, 2026–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing a sustainable pipeline of genetic medicines for rare disorders with high unmet need, today reported financial and recent operational results for the second quarter ended June 30, 2026.

“The second quarter of 2026 materially strengthened Rocket’s position, with the approval of KRESLADI demonstrating our ability to advance a genetic medicine through regulatory approval, the subsequent sale of the Priority Review Voucher generating $180 million in non-dilutive capital, and safety clearance for the first three Danon disease patients in our modified pivotal trial,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “Together with the more focused operating structure we implemented in late 2025, these achievements provide a stronger financial and executional foundation for advancing our cardiovascular pipeline. Based on the positive safety observations in Danon disease, we are working with the FDA on the path to treating additional patients and completing the pivotal trial as quickly as possible, while progressing toward additional regulatory and clinical milestones across the remainder of our cardiac portfolio, including PKP2-ACM and BAG3-DCM.”