Rocket Pharmaceuticals Reports Second Quarter 2024 Financial Results and Highlights Recent Progress Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase

Rocket Pharmaceuticals Reports Second Quarter 2024 Financial Results and

Highlights Recent Progress

Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat

PKP2 arrhythmogenic cardiomyopathy

Working toward FDA-approval of KRESLADI for severe LAD-I; Commercial infrastructure and capabilities in place for launch

Presented long-term KRESLADITM follow-up data from the global Phase 1/2 study for severe LAD-I, results from the global Phase 1/2 study of

RP-L102 for Fanconi Anemia, and data from the Phase 1 study of RP-L301 for PKD at ASGCT in May

Cash, cash equivalents and investments of approximately $278.8M; expected operational runway into 2026

CRANBURY, NJ - Aug. 5, 2024 - 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 quarter ending June 30, 2024.

"Over the quarter, Rocket has been advancing its clinical pipeline as we progressed our RP-A501 and RP-A601 cardiac programs, targeting Danon disease and PKP2-ACM, and

continued to actively enroll patients," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. "At ASGCT, we shared follow-up data from across our hematology portfolio including 4-year

follow-up data for KRESLADI to treat patients with severe LAD-I, demonstrating a 100% survival rate. In parallel, we have been preparing for the anticipated FDA approval of KRESLADI."

Recent Pipeline and Operational Updates

Second Quarter Financial Results

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies

designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living

with devastating rare diseases to experience long and full lives.

Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to

bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a

monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.

Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition

resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening

heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles.

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 other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on

these forward-looking statements, which often include words such as "could," "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of

those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte

Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory

interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and

clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its

product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation

that its cash, cash equivalents and investments will be sufficient to funds its operations into 2026. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales

and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop,

acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership

team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to

protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for

the year ended December 31, 2023, filed February 27, 2024 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such

statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.