Rocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million

About this update from Rocket Pharmaceuticals, Inc.

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 announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $180 million. The Rare Pediatric Disease Priority Review Voucher was granted by the FDA in March 2026 in connection with the approval of KRESLADI™, Rocket’s gene therapy for severe leukocyte adhesion deficiency-I (LAD-I), a rare and life-threatening primary immunodeficiency. As previously reported, before the PRV sale Rocket had cash, cash equivalents and investments of $144.4 million as of March 31, 2026. Following the $180 million in non-dilutive proceeds from the PRV sale, pro forma cash, cash equivalents and investments increased to approximately $322.6 million, which the Company expects to fund operations into the second quarter of 2028. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated biotechnology company advancing gene therapies for rare and devastating cardiovascular diseases, with additional programs in hematology and immunology. Rocket’s cardiovascular pipeline includes three clinical stage programs that each target one of the major inherited cardiomyopathy subtypes: hypertrophic, arrhythmogenic, and dilated cardiomyopathies. Together these conditions represent more than 100,000 patients in the U.S. and EU. The Company’s platform is supported by proprietary AAV manufacturing capabilities, multi-year efficacy and safety data in cardiac gene therapy, and experience treating several cardiac patients across late-stage AAV programs. For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and othe...

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