ROCKET PHARMACEUTICALS, INC.

Notes to Consolidated Financial Statements

($ in thousands, except shares and per share data) (Unaudited)

Nature of Business

Rocket Pharmaceuticals, Inc. (the “Company” or “Rocket”) is a fully integrated commercial-stage biotechnology company advancing a sustainable pipeline of genetic therapies, including one approved product and multiple investigational programs designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows the Company to design the optimal gene therapy for each indication, creating potential treatment options for people living with severe and rare diseases.

Rocket’sin vivo adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon disease, a devastating heart failure condition resulting in thickening of the heart, and early-stage clinical programs for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and BAG3-associated dilated cardiomyopathy (BAG3-DCM), a heart failure condition that causes enlarged ventricles.

Rocket’sex vivo lentiviral (LV) vector-based hematology portfolio includes KRESLADI™, which was granted FDA accelerated approval in March 2026 for severe Leukocyte Adhesion Deficiency-I (LAD-I), as well as additional programs for Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD).

Risks and Liquidity

The Company has not generated revenue from product sales to date and has incurred losses since inception. KRESLADI™ was approved by the FDA in March 2026 under the accelerated approval pathway; however, the Company has not yet generated product revenue. KRESLADI™ was approved under the FDA’s accelerated approval pathway based on an increase in neutrophil CD18 and CD11a surface expression. Continued approval may be contingent upon verification and description of clinical benefit through ongoing clinical follow-up and additional post-marketing data collection. Operations of the Company are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, having no commercial manufacturing experience, marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. Drug candidates currently under development will require significant additional R&D efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities. The commercialization of KRESLADI™ is subject to significant operational, logistical, and reimbursement-related risks. The delivery of autologous gene therapies requires complex coordination, including treatment center onboarding, manufacturing, and vein-to-vein logistics, which may impact the timing and scale of commercial uptake. In addition, given the ultra-rare patient population and anticipated phased commercial rollout, the Company does not expect KRESLADI™ to generate material revenue in the near term.

The Company’s product candidates are in the development and clinical stage. There can be no assurance that the Company’s R&D will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies.

In May 2025, two patients participating in the Phase 2 pivotal study of RP-A501 each experienced an unexpected SAE. Rocket voluntarily paused further Phase 2 study dosing in the U.S. and E.U., and the FDA placed a clinical hold on the trial to allow for further evaluation, which was lifted on August 20, 2025, after the Company satisfactorily addressed issues outlined in the clinical hold.

In July 2025, the Company implemented a strategic corporate reorganization designed to align its resources with its highest-priority programs, namely, the AAV-based gene therapy platform focused on cardiovascular diseases. As part of this effort, the Company reduced its workforce by approximately 30%.

The Company’s consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has incurred recurring losses and negative cash flows from operations and had an accumulated deficit of $1.49 billion as of March 31, 2026. As of March 31, 2026, the Company has $144.4 million of cash, cash equivalents, and investments. In April 2026, the Company entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million, which is expected to provide non-dilutive capital to support advancement of the Company’s cardiovascular gene therapy pipeline and extend its operational runway. Based on the Company’s current operating plan and the expected proceeds from the PRV sale, the Company believes its existing cash, cash equivalents, investments, and anticipated PRV proceeds will be sufficient to fund operations for at least twelve months from the filing date of this Quarterly Report on Form 10-Q.

In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company expects to continue to generate operating losses for the foreseeable future and to finance its future cash needs through, but not limited to, one or a combination of equity offerings, debt financings, collaborations, strategic partnerships and alliances or licensing arrangements. If the Company is unable to obtain funding, the Company would be forced to delay, reduce or eliminate some or all of its R&D programs, preclinical and clinical testing or commercialization efforts, which could adversely affect its business prospects. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies.

The Company may also face challenges retaining key personnel and maintaining continuity across teams, which could impair its ability to advance clinical programs, meet regulatory milestones, or pursue long-term strategic objectives. Potential litigation or other employee-related claims arising from the workforce reduction could divert management attention and further increase costs. Any of these factors could have a material adverse effect on our business, operating results, and financial condition.

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies