RCKT

Rocket Pharmaceuticals RCKT reported earnings of $1.08 per share (including certain one-time items) in the second quarter of 2026 in contrast to a loss of 62 cents per share incurred in the year-ago quarter. The Zacks Consensus Estimate for the company’s bottom line was earnings of 28 cents per share. Excluding these one-time items, the company reported a loss of 40 cents per share in the second quarter of 2026. Rocket Pharmaceuticals did not record any revenues in the second quarter of 2026. However, the Zacks Consensus Estimate for the company’s top line was $1 million. Year to date, shares of Rocket Pharmaceuticals have risen 3.7% compared with the industry’s increase of 6.1%. Image Source: Zacks Investment Research In the reported quarter, general and administrative expenses declined by around 30.4% year over year to $17.4 million, owing to lower legal expenses and other expenses. Research and development expenses were $29.5 million, down 30.9% from the year-ago quarter’s figure. The decrease was primarily driven by lower manufacturing and development costs and certain other costs. As of June 30, 2026, Rocket Pharmaceuticals had cash, cash equivalents and investments of $283.7 million compared with $144.4 million as of March 31, 2026. Management noted that the company’s current cash position reflects the proceeds from the sale of the Rare Pediatric Disease Priority Review Voucher. In March 2026, the FDA granted accelerated approval to RCKT’s gene therapy Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare genetic disorder. Following the nod, Kresladi became the first gene therapy to be approved by the FDA for treating children with severe LAD-I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. Per the company, launch preparations for Kresladi are underway, and patient onboarding is beginning in the fourth quarter of 2026. Rocket Pharmaceuticals is also developing an investigational gene therapy candidate, RP-A501, in a phase II study for treating patients with Danon disease. RP-A501 is the most advanced AAV-based candidate in the company’s pipeline. Earlier this month, the company announced positive initial safety findings from the first three patients treated with RP-A501 under a modified Phase 2 protocol for Danon disease. The patients received a recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg) with a refined immunomodulatory regimen, and no cases of thrombotic microangiopathy, capillary leak syndrome or other significant safety concerns were observed. RCKT is engaging with the FDA to determine the pathway for dosing additional patients and completing the pivotal phase II study, with an update on the regulatory pathway expected in the second half of 2026. Rocket Pharmaceuticals is also developing RP-A601 in an early-stage study for treating arrhythmogenic cardiomyopathy. The company also plans to start initial patient dosing in a phase I study evaluating another gene therapy candidate, RP-A701, for the treatment of dilated cardiomyopathy in the second half of 2026. Rocket Pharmaceuticals, Inc. price-consensus-eps-surprise-chart | Rocket Pharmaceuticals, Inc. Quote Rocket Pharmaceuticals currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Repligen RGEN and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.62 during the same time. RGEN shares have declined 0.4% year to date. Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%. Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 163.9% year to date. Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Rocket Pharmaceuticals, Inc. (RCKT) : Free Stock Analysis Report Repligen Corporation (RGEN) : Free Stock Analysis Report Liquidia Corporation (LQDA) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

All three initial patients treated with RP-A501 under the modified pivotal Phase 2 protocol completed the protocol-defined safety observation period; no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns observed Rocket is actively engaging with the FDA to align on the path to rapidly completing the pivotal Phase 2 trial in Danon disease Multiple clinical and regulatory milestones remain on track for the second half of 2026, including a broader Danon disease program update, a regulatory update for the planned RP-A601 pivotal study in PKP2-ACM, and initial patient dosing with RP-A701 in BAG3-DCM FDA approval of KRESLADI™ and subsequent monetization of the Priority Review Voucher generated $180 million in non-dilutive capital; focused preparations are underway for commercial availability and patient onboarding beginning in the fourth quarter of 2026 Cash, cash equivalents and investments of approximately $283.7 million; expected cash runway into the second quarter of 2028 CRANBURY, N.J., August 10, 2026--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and devastating diseases, focused on inherited cardiovascular disorders, today reported financial and recent operational results for the second quarter ended June 30, 2026. "The second quarter of 2026 materially strengthened Rocket’s position, with the approval of KRESLADI demonstrating our ability to advance a genetic medicine through regulatory approval, the subsequent sale of the Priority Review Voucher generating $180 million in non-dilutive capital, and safety clearance for the first three Danon disease patients in our modified pivotal trial," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "Together with the more focused operating structure we implemented in late 2025, these achievements provide a stronger financial and executional foundation for advancing our cardiovascular pipeline. Based on the positive safety observations in Danon disease, we are working with the FDA on the path to treating additional patients and completing the pivotal trial as quickly as possible, while progressing toward additional regulatory and clinical milestones across the remainder of our cardiac portfolio, including PKP2-ACM and BAG3-DCM." Recent Pipeline and Operational Updates Positive Initial RP-A501 Safety Findings Support Regulatory Engagement on Next Steps in Danon Disease Continued FDA Engagement and Phase 1 Enrollment for RP-A601 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) Phase 1 Trial Initiation Anticipated in 2026 for RP-A701 in BAG3-Associated Dilated Cardiomyopathy (BAG3-DCM) Focused Commercialization Strategy Underway for KRESLADI™ (marnetegragene autotemcel) Following FDA Approval Second Quarter 2026 Financial Results Cash position. Cash, cash equivalents and investments as of June 30, 2026, were $283.7 million. The cash position reflects the proceeds from the sale of the Rare Pediatric Disease Priority Review Voucher. R&D expenses. Research and development expenses were $29.5 million for the three months ended June 30, 2026, compared to $42.7 million for the three months ended June 30, 2025. The decrease of $13.2 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $7.5 million and stock-based and other compensation and benefit expenses of $3.6 million due to decreased R&D headcount, depreciation expenses of $1.0 million due to decreased asset base, and clinical trial expenses of $1.0 million. G&A expenses. General and administrative expenses were $17.4 million for the three months ended June 30, 2026, compared to $25.0 million for the three months ended June 30, 2025. The decrease of $7.6 million in G&A expenses was primarily driven by decreases in commercial preparation-related expenses of $4.8 million due to lower headcount and lower spending on commercial preparation, stock-based and other compensation and benefit expenses of $1.9 million due to decreased G&A headcount, and legal expenses of $1.4 million. Other Income. Other Income was $178.8 million for the three months ended June 30, 2026, compared to $2.2 million for the three months ended June 30, 2025. The increase in other income was primarily driven by the sale of the Rare Pediatric Disease Priority Review Voucher for net $178.2 million. The increase was partially offset by a decline in accretion of discount on investments, net, of $1.5 million due to lower investment balance and interest rates year over year. Income Taxes. Income taxes were $8.6 million for the three months ended June 30, 2026, compared to $0.0 million for the three months ended June 30, 2025, as a result of generating net income in the three months ended June 30, 2026. Net income. Net income was $123.2 million or $1.09 of basic income per share and $1.08 of diluted income per share for the three months ended June 30, 2026, compared to a net loss of $68.9 million or $0.62 (basic and diluted loss per share) for the three months ended June 30, 2025. Shares outstanding. 109,539,424 shares of common stock were outstanding as of June 30, 2026. Financial Guidance Cash position. As of June 30, 2026, Rocket had cash, cash equivalents and investments of $283.7 million. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of June 30, 2026, to fund operations into the second quarter of 2028. About KRESLADI™ KRESLADI™ (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 gene into a patient’s hematopoietic stem cells, enabling expression of CD18 and restoration of leukocyte adhesion and migration. KRESLADI 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. Severe 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. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Serious Infections Serious infections have occurred with KRESLADI administration. Increased susceptibility to infections may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of infection before and after KRESLADI infusion and treat appropriately. Administer prophylactic antimicrobials according to institutional guidelines. Avoid administration of KRESLADI in patients with active bloodstream infections or other serious, untreated infections. Any blood products required after KRESLADI infusion should be irradiated. Veno-Occlusive Disease Veno-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. Neutrophil Engraftment Failure Neutrophil engraftment failure may occur after treatment with KRESLADI. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 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. Delayed Platelet Engraftment Delayed platelet engraftment may occur after treatment with KRESLADI. Monitor platelet counts and bleeding until platelet engraftment and platelet recovery are achieved. LVV-Mediated Insertional Oncogenesis Lentiviral 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. Monitor 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. Hypersensitivity Reactions Hypersensitivity 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. Monitor 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. Anti-Retroviral Use Anti-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. Interference with Serology Testing Patients 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. Blood, Organ, Tissue, and Cell Donation Patients treated with KRESLADI should not donate blood, organs, tissues, or cells for transplantation at any time in the future. ADVERSE REACTIONS The most common non-laboratory adverse reactions (≥ 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. The most common laboratory adverse reactions (≥ 30% of patients) include: hemoglobin decreased, platelet count decreased, neutrophil count decreased, leukocyte count decreased, aspartate aminotransferase increased, and alanine aminotransferase increased. For additional safety information, refer to the full Prescribing Information. DRUG INTERACTIONS No 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. Vaccines The 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. Anti-retroviral Medications Patients 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. REFERENCE TO FULL PRESCRIBING INFORMATION Please see full Prescribing Information for KRESLADI. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated commercial-stage biotechnology company developing genetic medicines for rare and devastating diseases, with a strategic focus on inherited cardiovascular disorders and additional programs in hematology and immunology. Rocket's cardiovascular portfolio includes three clinical-stage gene therapy programs targeting hypertrophic, arrhythmogenic, and dilated cardiomyopathies, together representing one of the broadest pipelines focused on inherited heart disease. The Company's integrated platform combines proprietary AAV manufacturing capabilities, extensive clinical experience in cardiac gene therapy, and long-term safety and efficacy data supporting advancement across its cardiovascular portfolio. 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 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’s cash runway and financial position, Rocket’s planned use of proceeds from the monetization of the KRESLADI™ PRV, Rocket’s expectations of our ability to obtain additional funding to conduct our planned research and development efforts, the safety and effectiveness of RP-A501 in its pivotal Phase 2 trial under the modified protocol, the Company's ability to align with the FDA on the path to treating additional Danon disease patients, 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 cardiovascular AAV programs and KRESLADI™, including its planned pivotal trials, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket’s 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’s ongoing and planned clinical trials, 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, 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, our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring, including extending our cash runway, 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, 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260810430976/en/ Contacts Investors & Media Meg [email protected]

Shares of Rocket Pharmaceuticals RCKT were down on Friday after the company announced weaker-than-expected first-quarter 2026 earnings. RCKT incurred a loss of 42 cents per share in the first quarter of 2026, wider than the Zacks Consensus Estimate of a loss of 41 cents. In the year-ago quarter, the company had reported a loss of 56 cents per share. Rocket Pharmaceuticals did not record any revenues in the first quarter. Year to date, shares of Rocket Pharmaceuticals have risen 3.4% against the industry’s decline of 1.7%. Image Source: Zacks Investment Research In the reported quarter, general and administrative expenses declined by around 39.8% year over year to $17.1 million, owing to lower legal expenses and other expenses. Research and development expenses were $31.5 million, down 12.2% from the year-ago quarter’s figure. The decrease reflects more disciplined spending and resource management after the company’s recent organizational restructuring. As of March 31, 2026, Rocket Pharmaceuticals had cash, cash equivalents and investments of $144.4 million compared with $188.9 million as of Dec. 31, 2025. Management expects this cash balance, along with proceeds from the sale of the Priority Review Voucher (PRV) announced last month, to fund operations into the second quarter of 2028. In March 2026, the FDA granted accelerated approval to RCKT’s gene therapy Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare genetic disorder. Following the nod, Kresladi became the first gene therapy to be approved by the FDA for treating children with severe LAD-I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. With the FDA approval for Kresladi, the company received a Rare Pediatric Disease PRV, which is an incentive given by the FDA to encourage the development of drugs and biologics for rare and serious diseases. Last month, RCKT entered into a definitive agreement to sell its PRV for $180 million. The PRV monetization provides non-dilutive capital to support the company’s cardiovascular pipeline. Last August, the FDA lifted the clinical hold on the pivotal phase II study evaluating RCKT’s investigational gene therapy candidate, RP-A501, for treating patients with Danon disease. RP-A501 is the most advanced AAV-based candidate in the company’s pipeline. Per an agreement with the FDA, three more patients are being treated with a lower, recalibrated dose of RP-A501 (3.8 x 10¹³ GC/kg), with a minimum four-week interval between doses and an updated immune-modulating regimen. After these patients are treated, RCKT will discuss completion of the pivotal phase II study with the FDA. The company expects to provide an update in the second half of 2026 following these discussions. Rocket Pharmaceuticals is also developing RP-A601 in an early-stage study for treating arrhythmogenic cardiomyopathy. The company also plans to start clinical studies on a new gene therapy candidate, RP-A701, for the treatment of dilated cardiomyopathy by mid-2026. Rocket Pharmaceuticals, Inc. price-consensus-eps-surprise-chart | Rocket Pharmaceuticals, Inc. Quote Rocket Pharmaceuticals currently carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the biotech sector are Agenus AGEN, Amarin AMRN and AnaptysBio ANAB, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Over the past 60 days, estimates for Agenus’ 2026 earnings per share have risen from 58 cents to $2.10, while loss per share estimates for 2027 have narrowed from $1.51 to 73 cents during the same time. AGEN shares have surged 21.6% year to date. Agenus’ earnings beat estimates in two of the trailing four quarters, while missing the same on the remaining two occasions, with the average surprise being 31.42%. Over the past 60 days, 2026 loss per share estimates for Amarin have narrowed from $7.01 to $6.36, while the same for 2027 have narrowed from $5.50 to $4.64 during the same time. AMRN stock has risen 7.8% year to date. Amarin's earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 50.02%. Over the past 60 days, 2026 loss per share estimates for AnaptysBio have narrowed from $3.61 to $1.30, while estimates for 2027 have moved from a loss of $4.59 per share to earnings of $3.01 during the same time. ANAB stock has surged 43% year to date. AnaptysBio’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 59.70%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Agenus Inc. (AGEN) : Free Stock Analysis Report Amarin Corporation PLC (AMRN) : Free Stock Analysis Report AnaptysBio, Inc. (ANAB) : Free Stock Analysis Report Rocket Pharmaceuticals, Inc. (RCKT) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Pivotal Phase 2 trial of RP-A501 for Danon disease progressing with dosing reinitiated First patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026 KRESLADI™ granted FDA accelerated approval; Rare Pediatric Disease Priority Review Voucher monetized for $180 million Pro forma cash, cash equivalents and investments of approximately $322.6 million, including balance as of March 31, 2026, and non-dilutive proceeds from the PRV sale; expected operational runway into the second quarter of 2028 CRANBURY, N.J., May 07, 2026--(BUSINESS WIRE)--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 reported financial and recent operational results for the first quarter ended March 31, 2026. "During 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," said Gaurav Shah. "The 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." Recent Pipeline and Operational Updates Dosing the initial three-patient cohort for the Phase 2 study of RP-A501 in Danon disease is on track. Rocket previously disclosed that the U.S. Food and Drug Administration’s (FDA) lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months. Per agreement with FDA, three additional patients are being treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week inter-patient dosing interval and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with FDA regarding the completion of the Phase 2 pivotal study. Following FDA alignment, Rocket anticipates providing a program update in the second half of 2026. Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034. Engagement with FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM). Rocket continues to engage with FDA on alignment for a potential pivotal Phase 2 trial design for RP-A601 in PKP2-ACM, while the ongoing Phase 1 study remains open and actively enrolling to further characterize biological activity across a broader range of disease severity. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412. Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM). The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Dosing of the first patient is anticipated in mid-2026. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT07137338. FDA granted accelerated approval for KRESLADI™ (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). In March 2026, FDA granted KRESLADI™ accelerated approval for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. On April 28, 2026, Rocket announced a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million. First Quarter 2026 Financial Results Cash position. Cash, cash equivalents and investments as of March 31, 2026, were $144.4 million, excluding PRV monetization. R&D expenses. Research and development expenses were $31.5 million for the three months ended March 31, 2026, compared to $35.9 million for the three months ended March 31, 2025. The decrease of $4.4 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $5.8 million, stock-based and other compensation and benefit expenses of $2.0 million due to decreased R&D headcount, partially offset by increases in clinical trial expenses of $2.8 million and professional fees of $0.6 million. The reduction reflects disciplined resource allocation following the company’s recent organizational realignment. G&A expenses. General and administrative expenses were $17.1 million for the three months ended March 31, 2026, compared to $28.4 million for the three months ended March 31, 2025. The decrease of $11.3 million in G&A expenses was primarily driven by decreases in legal expenses of $5.7 million due to a legal settlement in 2025, decrease in commercial preparation-related expenses of $4.6 million due to lower headcount and lower spending on commercial launch, and stock-based and other compensation and benefit expenses of $2.8 million due to decreased G&A headcount, partially offset by an increase in milestone related expenses of $2.4 million. Net loss. Net loss was $47.6 million or $0.42 per share (basic and diluted) for the three months ended March 31, 2026, compared to $61.3 million or $0.56 (basic and diluted) for the three months ended March 31, 2025. Shares outstanding. 109,123,671 shares of common stock were outstanding as of March 31, 2026. Financial Guidance Cash position. As of March 31, 2026, Rocket had cash, cash equivalents and investments of $144.4 million. Rocket expects such resources, together with proceeds from the sale of the Priority Review Voucher, to be sufficient to fund its operations into the second quarter of 2028. About KRESLADI™ KRESLADI™ (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 gene into a patient’s hematopoietic stem cells, enabling expression of CD18 and restoration of leukocyte adhesion and migration. KRESLADI 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. Severe 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. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Serious Infections Serious infections have occurred with KRESLADI administration. Increased susceptibility to infections may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of infection before and after KRESLADI infusion and treat appropriately. Administer prophylactic antimicrobials according to institutional guidelines. Avoid administration of KRESLADI in patients with active bloodstream infections or other serious, untreated infections. Any blood products required after KRESLADI infusion should be irradiated. Veno-Occlusive Disease Veno-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. Neutrophil Engraftment Failure Neutrophil engraftment failure may occur after treatment with KRESLADI. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 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. Delayed Platelet Engraftment Delayed platelet engraftment may occur after treatment with KRESLADI. Monitor platelet counts and bleeding until platelet engraftment and platelet recovery are achieved. LVV-Mediated Insertional Oncogenesis Lentiviral 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. Monitor 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. Hypersensitivity Reactions Hypersensitivity 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. Monitor 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. Anti-Retroviral Use Anti-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. Interference with Serology Testing Patients 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. Blood, Organ, Tissue, and Cell Donation Patients treated with KRESLADI should not donate blood, organs, tissues, or cells for transplantation at any time in the future. ADVERSE REACTIONS The most common non-laboratory adverse reactions (≥ 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. The most common laboratory adverse reactions (≥ 30% of patients) include: hemoglobin decreased, platelet count decreased, neutrophil count decreased, leukocyte count decreased, aspartate aminotransferase increased, and alanine aminotransferase increased. For additional safety information, refer to the full Prescribing Information. DRUG INTERACTIONS No 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. Vaccines The 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. Anti-retroviral Medications Patients 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. REFERENCE TO FULL PRESCRIBING INFORMATION Please see full Prescribing Information for KRESLADI. 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 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’s cash runway and financial position, Rocket’s planned use of proceeds from the monetization of the KRESLADI™ PRV, Rocket’s expectations of our ability to obtain additional funding to conduct our planned research and development efforts, 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 cardiovascular AAV programs and KRESLADI™, including its planned pivotal trials, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket’s 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’s ongoing and planned clinical trials, 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, 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, our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring, including extending our cash runway, 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, 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260507193942/en/ Contacts Investors Meg Dodge [email protected] Media Kevin Giordano [email protected]

Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026 KRESLADI™ for severe LAD-I on track for March 28, 2026 PDUFA date Dosing of first patient in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026 Cash, cash equivalents and investments of approximately $188.9M; expected operational runway into the second quarter of 2027 CRANBURY, N.J., February 26, 2026--(BUSINESS WIRE)--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 fourth quarter and year ended December 31, 2025. "In 2025, we strengthened Rocket’s leadership in cardiovascular gene therapy, supported by more than five years of clinical experience in Danon disease and continued advancement across our PKP2-ACM and BAG3-DCM programs," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "Our focus remains on rigorous clinical execution across three cardiovascular programs, including resuming the Phase 2 pivotal clinical trial in Danon disease, initiating the Phase 1 clinical trial in BAG3-DCM, and advancing alignment with the FDA on the pivotal study design for PKP2-ACM. As we move through 2026, Rocket is well positioned to advance three high-impact cardiovascular programs and thoughtfully expand our cardiac gene therapy pipeline." Recent Pipeline and Operational Updates Dosing of additional patients for the Phase 2 study of RP-A501 for Danon disease anticipated in the first half of 2026. In August 2025, Rocket disclosed that the U.S. Food and Drug Administration’s (FDA) lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months. Per agreement with the FDA, three additional patients will be treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week interval between dosing and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with the FDA regarding the completion of the Phase 2 pivotal study. Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034. Engagement with the FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM). Rocket continues to work closely with the FDA to advance alignment on a potential pivotal Phase 2 trial design for RP-A601 in PKP2-ACM, while the ongoing Phase 1 study remains open and actively enrolling to further characterize biological activity across a broader range of disease severity. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412. Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM). The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Rocket expects to dose the first BAG3-DCM patient mid-2026. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT07137338. FDA accepted the resubmission of the BLA for KRESLADI™ (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). In October 2025, KRESLADI™ received a Prescription Drug User Fee Act (PDUFA) target action date for March 28, 2026. Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), with the approval of KRESLADI™. Fourth Quarter and Full Year 2025 Financial Results Cash position. Cash, cash equivalents and investments as of December 31, 2025, were $188.9 million. R&D expenses. Research and development expenses were $142.0 million for the twelve months ended December 31, 2025, compared to $171.2 million for the twelve months ended December 31, 2024. The decrease of $29.2 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $10.8 million, professional fees of $7.0 million, lab supplies and office expenses of $4.4 million, stock-based and other compensation and benefit expenses of $3.7 million, and clinical trial expenses of $2.7 million. The reduction reflects disciplined resource allocation following the company’s recent organizational realignment. G&A expenses. General and administrative expenses were $86.5 million for the twelve months ended December 31, 2025, compared to $102.0 million for the twelve months ended December 31, 2024. The decrease of $15.5 million in G&A expenses was primarily driven by decreases in commercial preparation-related expenses of $11.5 million from declining payroll and commercial launch services and stock-based and other compensation and benefit expenses of $5.4 million, partially offset by increase in legal expenses of $1.4 million. Net loss. Net loss was $223.1 million or $2.01 per share (basic and diluted) for the twelve months ended December 31, 2025, compared to $258.7 million or $2.73 (basic and diluted) for the twelve months ended December 31, 2024. Shares outstanding. 108,319,783 shares of common stock were outstanding as of December 31, 2025. Restructuring Expenses and Financial Guidance Restructuring expenses. Approximately $3.2 million in restructuring and restructuring-related charges were incurred in 2025. Cash position. As of December 31, 2025, Rocket had cash, cash equivalents and investments of $188.9 million. Rocket expects such resources, excluding any potential future proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADI™, will be sufficient to fund its operations into the second quarter of 2027. 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 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 an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. 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 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’s expectations of our ability to obtain additional funding to conduct our planned research and development efforts, 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 cardiovascular AAV programs and KRESLADI™, including its planned pivotal trials, and the safety, effectiveness and timing of related 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 its direct manufacturing capabilities for its AAV programs, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket’s 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, 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, 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, our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring, including extending our cash runway, and our estimates related to the costs and timing of implementing such initiatives, 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, 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20260226818001/en/ Contacts Investors Meg Dodge [email protected] Media Kevin Giordano [email protected]

Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026 KRESLADI™ for severe LAD-I on track for March 28, 2026 PDUFA date Leadership updates with the appointments of Dr. Syed Rizvi, Chief Medical Officer, Christopher Stevens, Chief Operating Officer, and Sarbani Chaudhuri, Chief Commercial & Medical Affairs Officer Cash, cash equivalents and investments of approximately $222.8M; expected operational runway into the second quarter of 2027 CRANBURY, N.J., November 06, 2025--(BUSINESS WIRE)--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 third quarter ending September 30, 2025. "During the third quarter, we maintained disciplined execution and sharpened our strategic focus on Rocket’s AAV cardiovascular gene therapy portfolio," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "In under three months, we aligned with the FDA to resume the Phase 2 pivotal study of RP-A501 for Danon disease, an important milestone that underscores the FDA’s collaborative approach and recognition of the urgent need for innovative therapies in rare cardiovascular disorders. In parallel, we are advancing RP-A601 toward a pivotal Phase 2 study in PKP2-ACM and preparing RP-A701 for first-in-human evaluation in BAG3-DCM. Finally, the upcoming March PDUFA date for KRESLADI™ represents a significant near-term commercial milestone as we work to bring this potentially life-saving therapy to patients with severe LAD-I." Recent Pipeline and Operational Updates Dosing of additional patients for the Phase 2 study of RP-A501 for Danon disease anticipated in the first half of 2026. In August, Rocket disclosed that the U.S. Food and Drug Administration’s (FDA) lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months. Per agreement with the FDA, three additional patients will be treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week interval between dosing and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with the FDA regarding the path forward for completion of the Phase 2 pivotal study. Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034. Engagement with the FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM). Rocket continues to collaborate with the FDA to align upon potential pivotal Phase 2 trial design to evaluate the efficacy and safety of RP-A601 for PKP2-ACM. Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM). The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT07137338. FDA accepted the resubmission of the BLA for KRESLADI™ (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). In October, KRESLADI™ received a Prescription Drug User Fee Act (PDUFA) target action date for March 28, 2026. Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), with the approval of KRESLADI™. Leadership updates with several key appointments to support Rocket’s transition toward late-stage development and anticipated commercialization. In September 2025, Syed Rizvi, M.D., was named Chief Medical Officer, adding over 20 years of clinical strategy, development and commercialization experience and a proven track record in cell and gene therapy, guiding the development and commercialization of multiple cell therapies for cancer treatments, including three approved autologous CAR-T cell therapies. Dr. Rizvi most recently served as Chief Medical Officer at Poseida Therapeutics, where he advanced the allogeneic CAR-T and gene therapy pipeline through the Company’s acquisition by Roche in January 2025. Previously, Dr. Rizvi held global clinical leadership roles at Legend Biotech, Celgene, Novartis, Merck, Caribou Biosciences and Genta Inc. Christopher Stevens, appointed Chief Operating Officer in July, adding more than two decades of global operations, product strategy, manufacturing, and supply chain leadership experience in the biopharmaceutical industry. Mr. Stevens previously served as Executive Vice President and Chief Patient Supply Officer at Spark Therapeutics, where he oversaw end-to-end gene therapy manufacturing and supply, and held senior operational roles at GlaxoSmithKline and Bristol Myers Squibb. Sarbani Chaudhuri, joined as Chief Commercial & Medical Affairs Officer in April, bringing more than 20 years of leadership in delivering multiple pioneering therapeutics from development to blockbuster launches that have transformed patient outcomes and been central to enterprise growth. Most recently, she led hematology at Johnson & Johnson, and previously held business leadership roles at AstraZeneca, Pfizer, and Novartis in oncology and rare diseases. Jonathan Schwartz, M.D., appointed Chief Science and Gene Therapy Officer, reflecting Rocket’s continued commitment to scientific and technical excellence as the Company advances toward late-stage development and commercialization. A founding member of Rocket’s leadership team and founding Chief Medical Officer, Dr. Schwartz has been instrumental in building and progressing Rocket’s industry-leading gene therapy pipeline across cardiology and hematology programs. In his expanded role, he will lead the strategic advancement and application of gene therapy technologies to current and future therapeutic areas, driving innovation across Rocket’s platform. Third Quarter 2025 Financial Results Cash position. Cash, cash equivalents and investments as of September 30, 2025, were $222.8 million. Rocket expects such resources, excluding any potential future proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADI™, will be sufficient to fund its operations into the second quarter of 2027. R&D expenses. Research and development expenses were $34.1 million for the three months ended September 30, 2025, compared to $42.3 million for the three months ended September 30, 2024. The decrease of $8.2 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $3.6 million, clinical trial expenses of $2.4 million, and professional fees of $2.0 million. The reduction reflects disciplined resource allocation following the company’s recent organizational realignment. G&A expenses. General and administrative expenses were $18.4 million for the three months ended September 30, 2025, compared to $27.1 million for the three months ended September 30, 2024. The decrease of $8.7 million in G&A expenses was primarily driven by decreases in commercial preparation-related expenses of $6.6 million and non-cash stock-based compensation expense of $1.5 million. Net loss. Net loss was $50.3 million or $0.45 per share (basic and diluted) for the three months ended September 30, 2025, compared to $66.7 million or $0.71 (basic and diluted) for the three months ended September 30, 2024. Shares outstanding. 108,208,643 shares of common stock were outstanding as of September 30, 2025. Restructuring Expenses and Financial Guidance Restructuring expenses. Approximately $3.3 million in restructuring and restructuring-related charges were incurred in 2025. 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 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 an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. 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 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 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 fund its operations into the second quarter of 2027. 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, 2024, filed February 27, 2025 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20251106225105/en/ Contacts Investors Meg Dodge [email protected] Media Kevin Giordano [email protected]

Prioritizing development of RP-A501 (Danon disease), RP-A601 (PKP2-ACM), and RP-A701 (BAG3-DCM) to advance AAV cardiovascular gene therapy platform for sustained value creation FDA RMAT designation awarded to RP-A601 for PKP2-ACM; Engaging with FDA on pivotal trial design following encouraging initial Phase 1 data at ASGCT IND accepted and FDA Fast Track designation received for RP-A701 in BAG3-DCM program; Phase 1 trial start-up activities underway Organizational restructuring expected to reduce headcount by approximately 30% and lower 12-month cash burn by nearly 25% Chris Stevens appointed Chief Operating Officer Cash, cash equivalents and investments of approximately $271.5M; expected operational runway into the second quarter of 2027 CRANBURY, N.J., August 07, 2025--(BUSINESS WIRE)--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 second quarter ending June 30, 2025. "The second quarter of 2025 marked an important inflection point for Rocket as we refined our strategic focus around our AAV cardiovascular gene therapy platform and took multiple decisive steps to strengthen our financial foundation and thoughtfully adjust to market dynamics. We are fortifying our path to sustained value creation by leaning into programs with the highest value, conserving cash, and driving an efficient, agile organization," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "With regulatory alignment to resolve the clinical hold for RP-A501 in Danon disease in progress, momentum building for RP-A601 in PKP2-ACM as it advances toward a pivotal Phase 2 trial, and RP-A701 preparing to enter the clinic for the treatment of BAG3-DCM, Rocket is positioned as the leader in the development of gene therapies for inherited cardiomyopathies. While we are pausing additional investments in the FA and PKD programs, we are deeply grateful and committed to the patient communities and are exploring strategic alternatives to advance these programs externally. Finally, our recent reorganization ensures we are appropriately resourced to execute on our near-term milestones with nearly two years of capital." Recent Pipeline and Operational Updates Investigation into the Serious Adverse Event (SAE) from the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing. Rocket previously disclosed an SAE, related to clinical complications from capillary leak syndrome and unfortunately leading to the patient’s death. In response, Rocket paused dosing and initiated a root cause analysis, focusing on the recent addition of a C3 inhibitor to the pre-treatment regimen. On May 23, 2025, FDA placed the trial on clinical hold for further evaluation. Rocket is actively working with FDA, independent safety monitors, and clinical experts to ensure patient safety and resume the trial. While the clinical hold remains in place, the company cannot provide guidance on trial completion timing. Actively engaging with FDA on the advancement of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) in potential pivotal trial following initial positive Phase 1 data at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). In July, Rocket received FDA Regeneration Medicine Advanced Therapy (RMAT) designation for RP-A601 for PKP2-ACM. Initial Phase 1 data demonstrating encouraging safety and preliminary efficacy outcomes of RP-A601 for PKP2-ACM were presented at ASGCT in May 2025. Rocket is engaging with FDA on potential pivotal trial design to evaluate the efficacy and safety of RP-A601. Phase 1 trial start-up activities are underway for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM). In June, an Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-DCM, received clearance from FDA. RP-A701 was recently granted FDA Fast Track designation, designed to facilitate the development and expedite the review of therapies for serious or life-threatening conditions that fill an unmet medical need. This enables increased communication with FDA, the potential for accelerated approval, and permits a rolling Biologics License Application (BLA) review. The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. BAG3-DCM is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality. Rocket estimates that the prevalence of BAG3-DCM in the U.S. is as many as 30,000 individuals. FDA review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). Rocket previously disclosed that FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I. The Company continues to work with senior leaders and reviewers from FDA’s Center for Biologics Evaluation and Research and submission of complete BLA to resolve Complete Response Letter is anticipated before the end of 2025. Ongoing strategic corporate restructuring and pipeline prioritization. As part of its broader strategic reorganization, Rocket implemented a workforce reduction of approximately 30%, across all functions, to align with a pipeline prioritization plan focused exclusively on its AAV cardiovascular gene therapy platform. This restructuring, together with other cost-saving measures, is expected to reduce operating expenses by nearly 25% over the next 12 months. The reorganization enables the company to focus on its late‑stage AAV gene therapy programs in Danon disease, PKP2‑ACM, and BAG3‑DCM, while advancing regulatory activities for KRESLADI™ in severe LAD‑I. As part of this realignment, Rocket is pausing additional investments in its Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs. The company continues to evaluate options to advance the FA program with health authorities in alignment with its refined strategic focus and broader corporate priorities. In July, Rocket appointed Chris Stevens as Chief Operating Officer. Chris Stevens is a highly seasoned executive with 25 years of experience across technical operations, product strategy, and general management, bringing immense commercial technical operations experience to Rocket. Stevens most recently served as the Executive Vice President and Chief Patient Supply Officer at Spark Therapeutics (subsidiary of Roche), where he successfully led teams across manufacturing, supply chain, quality, compliance, engineering, EHS, and facilities management, playing a key role in delivering gene therapies to patients. Second Quarter 2025 Financial Results Cash position. Cash, cash equivalents and investments as of June 30, 2025, were $271.5 million. Rocket expects such resources, excluding any potential proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADITM, will be sufficient to fund its operations into the second quarter of 2027. R&D expenses. Research and development expenses were $42.7 million for the three months ended June 30, 2025, compared to $46.3 million for the three months ended June 30, 2024. The decrease of $3.7 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $2.3 million, professional fees of $2.0 million and building supplies and consumables of $0.8 million, offset by an increase in clinical trial expenses of $1.2 million. G&A expenses. General and administrative expenses were $25.0 million for the three months ended June 30, 2025, compared to $27.4 million for the three months ended June 30, 2024. The decrease in G&A expenses was primarily driven by decreases in commercial preparation related expenses of $1.4 million and compensation and benefits expense of $0.9 million. Net loss. Net loss was $68.9 million or $0.62 per share (basic and diluted) for the three months ended June 30, 2025, compared to $69.6 million or $0.74 (basic and diluted) for the three months ended June 30, 2024. Shares outstanding. 107,884,420 shares of common stock were outstanding as of June 30, 2025. Restructuring Expenses and Financial Guidance Restructuring expenses. Approximately $3.5 million in restructuring and restructuring-related charges were incurred in the first half of 2025. 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 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 an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. 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 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 ability to realize the intended benefits of the restructuring plan and reduction in workforce, 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 fund its operations into the second quarter of 2027. There can be no assurance that the restructuring plan or the planned reduction in workforce will have the intended effect on the Company’s operational results and strategic decisions, that any anticipated charges and any anticipated cost savings associated with the restructuring plan or the reduction in workforce will achieve their intended benefits. 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, 2024, filed February 27, 2025 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20250807231064/en/ Contacts Investors Meg Dodge [email protected] Media Kevin Giordano [email protected]