Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results Advanced pipeline of four clinical programs across AAV cardiology and LV hematology portfolios delivering strong results

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results

Advanced pipeline of four clinical programs across AAV cardiology and LV hematology portfolios delivering strong results for Danon

Disease, Fanconi Anemia, LAD-I and PKD

Expanded leadership position in AAV cardiac gene therapy, with significant opportunities for value creation across Danon Disease,

PKP2-ACM and BAG3-DCM programs

Established in-house cGMP manufacturing for AAV, poised to support Danon pivotal trial in Q2'23 and subsequent commercialization

Cash, cash equivalents and investments of approximately $400M; expected operational runway through 2024

CRANBURY, N.J. - Feb. 27, 2023 - Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and

sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022.

"Rocket closed 2022 with positive results across four clinical gene therapy programs spanning both our AAV cardiology and LV hematology therapeutic areas in an

unprecedented fashion for a gene therapy company, validating our approach to strong science and R&D, carefully selected assets and smart execution. We are thrilled to begin 2023 with the FDA recently granting RMAT designation to RP-A501 for

Danon Disease, validating the strength of our results and underscoring RP-A501's potential as a transformative therapy for Danon patients," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.

Dr. Shah continued, "At the same time, we expanded our leadership position in AAV cardiac gene therapy with the recent unveiling of compelling preclinical proof of

concept for RP-A601 for the treatment of PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) and addition of the BAG3-associated dilated cardiomyopathy (DCM) asset following the acquisition of Renovacor. Taken together, these two diseases along with

Danon Disease affect more than 100,000 patients in the U.S. and EU. Further, our LV hematology portfolio delivered stellar results as we now advance towards our first regulatory filings for Leukocyte Adhesion Deficiency (LAD-I) in the second

quarter of 2023 and Fanconi Anemia (FA) in the fourth quarter of 2023, and subsequent commercialization."

"I am also pleased that we brought in additional funds of $197.7 million in 2022. We begin this year in a strong financial position to execute on near and long-term

milestones, anticipating that our cash runway of approximately $400 million will fund operations through 2024," said Dr. Shah. "I am grateful to our growing Rocket team of multi-disciplinary experts, scientific collaborators and the critical voices

of the patient community who were essential to this progress and continue to help advance our mission of seeking gene therapy cures. I look forward to building off the successes of 2022 and continuing our progress in 2023."

Key Pipeline and Operational Updates

Upcoming Investor Conferences

Fourth Quarter and Full Year 2022 Financial Results

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of

complex and rare disorders. The Company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical

programs using lentiviral vector (LV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure 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 rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to

life-threatening anemia. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has preclinical AAV-based gene therapy programs in

PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated

cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, 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, Rocket's plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the

safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other

federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "aim," "anticipate," "believe," "can," "continue,"

"design," "estimate," "expect," "intend," "may," "plan," "potential," "will give," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. 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 ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket's ongoing

trials, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical

studies and clinical trials of its product candidates, our ability to submit regulatory filings with the U.S. Food and Drug Administration (FDA) and to obtain and maintain FDA or other regulatory authority approval of our product candidates, Rocket's

dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, our competitors' activities, including decisions as to the timing of competing product launches, pricing and

discounting, our integration of an acquired business, which involves a number of risks, including the possibility that the integration process could result in the loss of key employees, the disruption of our ongoing business, or inconsistencies in

standards, controls, procedures, or policies, our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire and any unexpected expenditures, 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, 2021, filed February 28, 2022 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.