ROCKET PHARMACEUTICALS, INC.

Highlights Recent Progress

Pivotal Phase 2 trial of RP-A501 for Danon disease progressing with dosing reinitiated

First patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy

anticipated in mid-2026

KRESLADI™ granted FDA accelerated approval; Rare Pediatric Disease Priority Review Voucher

monetized for $180 million

Pro forma cash, cash equivalents and investments of approximately $322.6 million, including balance as of March 31, 2026, and non-dilutive proceeds from the PRV sale; expected operational runway into the second quarter of 2028

CRANBURY, NJ–May 7, 2026–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-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 first quarter ended March 31, 2026.

“During the first quarter, we continued advancing our cardiovascular gene therapy portfolio, including reinitiating dosing in the initial three-patient cohort of our pivotal Phase 2 study of RP-A501 for Danon disease, while also achieving the accelerated approval of KRESLADI for severe LAD-I,” said Gaurav Shah. “The subsequent $180 million non-dilutive monetization of our Rare Pediatric Disease Priority Review Voucher further strengthened our balance sheet and extended our expected cash runway into the second quarter of 2028. We remain focused on disciplined execution across our deep cardiovascular pipeline and anticipate providing an update on the Danon program in the second half of the year.”