\n\u201cDuring 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,\u201d said Gaurav Shah. \u201cThe 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.\u201d<\/p>\n
Recent Pipeline and Operational Updates<\/b><\/p>\n First Quarter 2026 Financial Results<\/b><\/p>\n Financial Guidance<\/b><\/p>\n About KRESLADI<\/b>\u2122<\/b><\/p>\n \nKRESLADI\u2122 (marnetegragene autotemcel) is an autologous hematopoietic stem cell-based gene therapy designed to address the underlying genetic cause of severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare, life-threatening pediatric immunodeficiency. The therapy utilizes ex vivo lentiviral vector-mediated gene transfer to introduce a functional copy of the ITGB2<\/i> gene into a patient\u2019s hematopoietic stem cells, enabling expression of CD18 and restoration of leukocyte adhesion and migration.<\/p>\n \nKRESLADI is administered as a one-time intravenous infusion following myeloablative conditioning. In clinical studies, treatment with KRESLADI resulted in increased neutrophil CD18 and CD11a surface expression, supporting the biological activity of the therapy and forming the basis for accelerated approval. Continued approval may be contingent upon verification and description of clinical benefit through ongoing clinical follow-up and additional post-marketing data collection.<\/p>\n \nSevere LAD-I is characterized by recurrent, serious bacterial and fungal infections beginning in early infancy and is associated with high early-childhood mortality without effective treatment.<\/p>\n IMPORTANT SAFETY INFORMATION<\/b><\/p>\n WARNINGS AND PRECAUTIONS<\/b><\/p>\n Serious Infections<\/b><\/p>\n \nSerious infections have occurred with KRESLADI administration. Increased susceptibility to infections may occur due to administration of myeloablative conditioning prior to KRESLADI infusion.<\/p>\n \nMonitor patients for signs and symptoms of infection before and after KRESLADI infusion and treat appropriately. Administer prophylactic antimicrobials according to institutional guidelines.<\/p>\n \nAvoid administration of KRESLADI in patients with active bloodstream infections or other serious, untreated infections.<\/p>\n \nAny blood products required after KRESLADI infusion should be irradiated.<\/p>\n Veno-Occlusive Disease<\/b><\/p>\n \nVeno-occlusive disease has occurred with KRESLADI treatment. Increased susceptibility to veno-occlusive disease may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of veno-occlusive disease including assessment of liver function tests during the first month following KRESLADI infusion.<\/p>\n Neutrophil Engraftment Failure<\/b><\/p>\n \nNeutrophil engraftment failure may occur after treatment with KRESLADI. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) \u2265 500 cells\/microliter obtained on different days by Day 43 after infusion of KRESLADI. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with KRESLADI, provide rescue treatment with the back-up collection of CD34+ cells.<\/p>\n Delayed Platelet Engraftment<\/b><\/p>\n \nDelayed platelet engraftment may occur after treatment with KRESLADI. Monitor platelet counts and bleeding until platelet engraftment and platelet recovery are achieved.<\/p>\n LVV-Mediated Insertional Oncogenesis<\/b><\/p>\n \nLentiviral vector (LVV)-mediated insertional oncogenesis may occur after treatment with KRESLADI. Hematologic malignancy is a lifelong risk and patients treated with KRESLADI may develop hematologic malignancy at any time following treatment.<\/p>\n \nMonitor for hematologic malignancies clinically, and with a complete blood count (with differential) at least annually and integration site analysis as warranted for at least 15 years after treatment with KRESLADI and as clinically indicated. If malignancy is detected in any patient who received KRESLADI, contact Rocket Pharmaceuticals, Inc. at 1-800-982-2410 for reporting and to obtain instructions on collection of samples for testing.<\/p>\n Hypersensitivity Reactions<\/b><\/p>\n \nHypersensitivity reactions including anaphylaxis may occur with the infusion of KRESLADI. The dimethyl sulfoxide (DMSO) in KRESLADI may cause hypersensitivity reactions which may occur in patients with and without prior exposure to DMSO.<\/p>\n \nMonitor patients for signs and symptoms of hypersensitivity reactions during and after KRESLADI infusion. If a hypersensitivity reaction occurs, pause infusion if ongoing and manage according to clinical practice.<\/p>\n Anti-Retroviral Use<\/b><\/p>\n \nAnti-retroviral medications may interfere with manufacturing of KRESLADI. If a patient requires anti-retrovirals for HIV prophylaxis, mobilization and apheresis of CD34+ cells for KRESLADI manufacturing should be delayed until HIV infection is adequately ruled out. Patients should not take anti-retroviral medications for at least one month prior to mobilization, or for the expected duration required for the elimination of the anti-retroviral medications, and until all cycles of apheresis are completed.<\/p>\n Interference with Serology Testing<\/b><\/p>\n \nPatients who have received KRESLADI are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to LVV provirus insertion resulting in a false-positive test for HIV. Therefore, patients who have received KRESLADI should not be screened for HIV infection using a PCR-based assay.<\/p>\n Blood, Organ, Tissue, and Cell Donation<\/b><\/p>\n \nPatients treated with KRESLADI should not donate blood, organs, tissues, or cells for transplantation at any time in the future.<\/p>\n ADVERSE REACTIONS<\/b><\/p>\n \nThe most common non-laboratory adverse reactions (\u2265 30% of patients) include: mucositis, upper respiratory tract infection, viral infection, febrile neutropenia, skin lesion, nausea\/vomiting, rash\/dermatitis, pyrexia, device related infection, and skin infection.<\/p>\n \nThe most common laboratory adverse reactions (\u2265 30% of patients) include: hemoglobin decreased, platelet count decreased, neutrophil count decreased, leukocyte count decreased, aspartate aminotransferase increased, and alanine aminotransferase increased.<\/p>\n \nFor additional safety information, refer to the full Prescribing Information<\/a>.<\/p>\n DRUG INTERACTIONS<\/b><\/p>\n \nNo formal drug interaction studies have been performed. KRESLADI is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters.<\/p>\n Vaccines<\/b><\/p>\n \nThe safety and effectiveness of immunization with live viral vaccines during or following KRESLADI treatment has not been studied. Vaccination is not recommended during the 6 weeks preceding the start of myeloablative conditioning, and until hematological recovery following treatment with KRESLADI. Where feasible, administer childhood vaccinations prior to myeloablative conditioning for KRESLADI.<\/p>\n Anti-retroviral Medications<\/b><\/p>\n \nPatients should not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization and for the expected duration for elimination of the medications, and until all cycles of apheresis are completed. Anti-retroviral medications may interfere with manufacturing of KRESLADI.<\/p>\n REFERENCE TO FULL PRESCRIBING INFORMATION<\/b><\/p>\n \nPlease see full Prescribing Information<\/a> for KRESLADI.<\/p>\n About Rocket Pharmaceuticals, Inc.<\/b><\/p>\n \nRocket 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\u2019s 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\u2019s 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<\/a> and follow us on LinkedIn<\/a>, YouTube<\/a>, and X<\/a>.<\/p>\n Rocket Cautionary Statement Regarding Forward-Looking Statements<\/b><\/p>\n \nThis press release contains forward-looking statements concerning Rocket\u2019s 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 “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\u2019s cash runway and financial position, Rocket\u2019s planned use of proceeds from the monetization of the KRESLADI\u2122 PRV, Rocket\u2019s expectations of our ability to obtain additional funding to conduct our planned research and development efforts, the expected timing and data readouts of Rocket\u2019s ongoing and planned clinical trials, the expected timing and outcome of Rocket\u2019s regulatory interactions and planned submissions, Rocket\u2019s plans for the advancement of its cardiovascular AAV programs and KRESLADI\u2122, including its planned pivotal trials, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, Rocket\u2019s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket\u2019s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies. 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, the results of Rocket\u2019s ongoing and planned clinical trials, Rocket\u2019s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket\u2019s competitors\u2019 activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket\u2019s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, Rocket\u2019s 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, our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring, including extending our cash runway, Rocket\u2019s 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\u2019s Annual Report on Form 10-K for the year ended December 31, 2025, filed February 26, 2026 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.<\/p>\n \n\u00a0<\/p>\n<\/td>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n31,454<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n35,942<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n17,057<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n28,446<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n48,511<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n64,388<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(48,511<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(64,388<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(473<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(472<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n161<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,336<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n1,229<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n2,190<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(47,594<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(61,334<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(0.42<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(0.56<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n112,134,059<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n110,093,461<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n144,379<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n188,929<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n285,407<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n330,449<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n47,368<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n53,228<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n238,039<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n277,221<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \n\u00a0<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20260507193942\/en\/<\/a><\/span><\/p>\n Investors Media KEYWORDS:<\/b> New Jersey United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Health Genetics Clinical Trials Pharmaceutical Cardiology Biotechnology<\/p>\n\n
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\n \n \n Three Months Ended March 31,<\/b><\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \n \n Operating expenses:<\/td>\n \n \n \n <\/tr>\n \n Research and development<\/td>\n \n \n \n \n \n \n \n \n \n General and administrative<\/td>\n \n \n \n \n \n \n \n \n \n Total operating expenses<\/td>\n \n \n \n \n \n \n \n \n \n Loss from operations<\/td>\n \n \n \n \n \n \n \n \n \n Interest expense<\/td>\n \n \n \n \n \n \n \n \n \n Interest and other income, net<\/td>\n \n \n \n \n \n \n \n \n \n Accretion of discount on investments, net<\/td>\n \n \n \n \n \n \n \n \n \n Net loss<\/td>\n \n \n \n \n \n \n \n \n \n Net loss per share – basic and diluted<\/td>\n \n \n \n \n \n \n \n \n \n Weighted-average common shares outstanding – basic and diluted<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n March 31, 2026<\/b><\/td>\n \n December 31, 2025<\/b><\/td>\n<\/tr>\n \n Cash, cash equivalents, and investments<\/td>\n \n \n \n \n \n \n \n \n \n Total assets<\/td>\n \n \n \n \n \n \n \n \n \n Total liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Total stockholders’ equity<\/td>\n \n \n \n \n \n \n \n \n <\/p>\n
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Meg Dodge
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mdodge@rocketpharma.com<\/a><\/p>\n
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Kevin Giordano
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media@rocketpharma.com<\/a><\/p>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20260507193942\/en\/1151092\/3\/Rocket_Pharma.jpg\")
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