Marnetegragene autotemcel

有一种病，患者人数可能只有几万、几千，甚至几百人。 药厂不愿意研发，因为赚不到钱；医生没有经验，因为一辈子见不到几例；患者家庭四处求医，最终得到的答案往往是同一句："这个病，没有药。" 它们被称为 "孤儿病"—— 医学体系里被遗忘的角落。 但 2026 年，这个角落正在被一束光照亮。短短 8 个月内，FDA 密集批准了 4 款罕见病基因疗法，覆盖代谢病、耳聋、免疫缺陷、血液病四个完全不同的领域。每一款的背后，都是一个曾经 "无药可治" 的群体，第一次看到了 "一次治疗、终身纠正" 的可能。 而推动这一切加速的，除了基因治疗技术的成熟，还有一个越来越重要的角色 ——AI。4 款疗法，4 个群体的命运转折1. Genglycos：让糖原贮积症患儿告别 "半夜喂玉米淀粉" 8 月 19 日，FDA 加速批准 Genglycos（DTX401），用于 8 岁及以上 Ia 型糖原贮积症（GSDIa）。这是全球首个针对该病的基因疗法。 Ia 型糖原贮积症，听起来陌生，但对患者家庭来说，是一场每天都在进行的战斗。 G6PC 基因突变导致肝脏无法正常分解糖原。患者从婴儿期开始，就会反复出现严重低血糖 —— 如果夜里不按时进食，可能在睡梦中陷入昏迷、抽搐，甚至死亡。 为了维持血糖，患儿需要每 3-4 小时吃一次生玉米淀粉，包括半夜。很多家长定着闹钟，凌晨 2 点、5 点起来给孩子喂粉，一喂就是十几年。长期代谢紊乱还会导致肝肿大、高尿酸、高脂血症，成年后肝癌和肾衰竭的风险显著升高。 Genglycos 的逻辑很直接：用 AAV 病毒载体，把正常的 G6PC 基因递送到肝脏，让肝细胞重新获得分解糖原的能力。一次输注，理论上终身有效。患者有望告别夜间喂食和严格饮食限制，像正常人一样生活。 这款药由 Ultragenyx 研发，从递交申请到获批走的是加速审批通道 —— 因为对于这种致命的罕见病，患者等不起。2. Otarmeni：从全聋到听见耳语，只用了一针 4 月 23 日，FDA 批准 Otarmeni（DB-OTO），用于 OTOF 基因双等位变异导致的重度至极重度感音神经性耳聋。这是全球首个双 AAV 载体基因疗法。 更惊人的是审批速度：从递交生物制品许可申请到正式获批，仅用了 61 天，创下 FDA 历史上基因疗法最快审批纪录。 为什么这么快？因为临床数据太有说服力了。 OTOF 基因编码耳畸蛋白，是内耳毛细胞把声音振动转化为神经电信号的关键 "转换器"。这个基因坏了，孩子出生就是全聋，人工耳蜗也无法解决根本问题 —— 因为信号转换的那一步断了。 Otarmeni 通过双 AAV 载体（因为 OTOF 基因太大，一个 AAV 装不下，需要拆成两段递送到同一个细胞里重新组装），把正常基因送进内耳毛细胞。 《新英格兰医学杂志》发表的试验数据显示：治疗后，多名原本全聋的儿童恢复了听力，部分患儿能听到耳语级别的声音，言语发育能力接近正常同龄儿童。 一个从出生就活在无声世界里的孩子，一针之后，第一次听见了妈妈的声音。 这不仅是一款药的获批，更是人类首次证明：基因疗法可以恢复听觉功能。全球约一半的先天性耳聋由单基因突变导致，OTOF 只是第一个，后续更多亚型的耳聋基因疗法已经在路上。3. Kresladi：百万分之一的孩子，终于有了不用等供体的治疗 3 月 26 日，FDA 批准 Kresladi，用于 ITGB2 基因突变导致的重度白细胞黏附缺陷症 I 型（LAD-1），且无匹配同胞供体的患儿。这是全球首个 LAD-1 基因疗法。 LAD-1 的发病率约为百万分之一。 ITGB2 基因突变后，白细胞表面缺少一种叫 CD18 的蛋白，导致白细胞无法从血管里 "爬出去" 到达感染部位。结果就是：身体有感染，但白细胞过不去。 患儿出生后就反复发生严重细菌感染 —— 皮肤溃烂不愈合、牙周组织坏死、肺炎、败血症。很多孩子在婴幼儿期就死于感染，能活过青春期的极少。 此前唯一的根治手段是造血干细胞移植，但需要找到匹配的供体。对于这种百万分之一的病，找到匹配供体的概率微乎其微。 Kresladi 的做法是：取出患者自己的造血干细胞，在体外用慢病毒载体插入正常的 ITGB2 基因，然后回输。不需要供体，不会排异，一次治疗修复免疫系统。 百万分之一的概率，终于等来了百万分之一的希望。4. Casgevy：把治疗年龄降到 2 岁，在器官损坏之前拦住疾病 7 月 1 日，FDA 批准 Casgevy 扩展适应症，用于 2 岁及以上镰状细胞病或输血依赖型 β 地中海贫血患者。这是首个获批用于 2 岁以上儿童的镰状细胞病基因疗法。 镰状细胞病可能是这 4 种病里读者最熟悉的。患者的红细胞会变成镰刀状，堵塞血管，引发剧烈疼痛、卒中、急性胸部综合征、器官损伤。儿童期就可能发生卒中，很多患者活不到 40 岁。 Casgevy 是全球首款 CRISPR 基因编辑疗法，通过编辑患者自身造血干细胞的 BCL11A 基因，重新激活胎儿血红蛋白的表达，让红细胞恢复正常形态。 此前 Casgevy 已获批用于 12 岁以上患者。这次扩展到 2 岁，意义远不止 "年龄放宽"—— 它意味着可以在器官发生不可逆损伤之前就干预。 镰状细胞病的卒中、脾损伤、肺损伤很多在儿童期就开始累积，等到 12 岁再治疗，有些损伤已经无法挽回。2 岁开始治疗，从 "挽救生命" 升级为 "预防残疾"。为什么是 2026 年？基因疗法的 "平台红利期" 到了 4 款疗法在 8 个月内密集获批，不是巧合，而是技术平台积累到了临界点。 第一，AAV 载体技术成熟了。腺相关病毒（AAV）是目前最主流的基因递送载体。过去十年，科学家解决了 AAV 的靶向性、免疫原性、装载容量三大问题。Otarmeni 用的双 AAV 技术，能把超过 4.7kb 的大基因（OTOF）拆成两段递送到同一个细胞内完成重组 —— 这在 5 年前还是不可想象的。 第二，"一次治愈" 的商业模式被验证了。基因疗法定价高昂（通常在百万美元级别），但 "一次给药、终身有效" 的逻辑正在被医保和支付方接受。Casgevy 在美国定价 220 万美元，但与终身输血、排铁、住院的费用相比，长期来看反而更经济。支付模式的跑通，让药企愿意投入罕见病基因疗法。 第三，监管通道更顺畅了。FDA 的加速批准、罕见儿科疾病优先审评券、再生医学先进疗法（RMAT）认定，这些通道叠加，让罕见病基因疗法的审批速度大幅提升。Otarmeni 的 61 天获批，就是多重通道叠加的结果。AI 正在改写罕见病的 "经济学" 基因疗法解决了 "怎么治" 的问题，而 AI 正在解决更前端的问题：治什么？ 罕见病药物研发的核心困境是 "三少"：患者少（临床试验招募难）、数据少（基础研究不足）、利润少（药企投入意愿低）。全球约 4.46 亿人受罕见病影响，但不到 5% 的罕见病有获批治疗。 AI 恰好能在这三个维度上破局：1. 靶点发现：从 "无结构" 到 "可成药" 很多罕见病相关蛋白，因为表达量低、不稳定、膜蛋白等原因，传统实验方法（X 射线晶体学、冷冻电镜）很难解析结构。不知道蛋白长什么样，就找不到药物结合的 "口袋"，也就无法设计药物。 AlphaFold 改变了这一切。2026 年 3 月，Google DeepMind 与华盛顿大学团队在《The Lancet Digital Health》发表研究，用 AlphaFold 为 3 种此前完全没有结构数据的罕见遗传病绘制了蛋白质三维图谱，识别出导致细胞功能异常的结构缺陷，并启动了 FDA 已批药物的虚拟筛选 —— 也就是 "老药新用"。 这些病因为患者太少，传统药企根本不会投入资源去解析蛋白结构。AI 用几小时的计算，替代了过去几年的实验工作，把 "不可能研究的病" 变成了 "可以找药的病"。2. 药物重定位：跳过 10 年研发周期 对于罕见病来说，从零研发新药几乎不现实 —— 一个新药平均需要 12 年、26 亿美元。但如果能从已经获批的药物里筛选出对罕见病有效的 "老药"，就可以跳过安全性验证的漫长过程，直接进入 II 期概念验证。 AI 驱动的虚拟筛选，可以在几天内完成数十亿化合物的对接模拟，找到可能结合缺陷蛋白的已有药物。这对于患者只有几百人的超罕见病来说，可能是唯一可行的研发路径。3. 临床产出：AI 药物开始进入人体试验 根据波士顿咨询集团（BCG）2026 年 5 月的追踪分析，全球已有超过 75 个由 AI 设计或深度参与的药物分子进入活跃的人体临床试验。 2026 年 6 月，Google DeepMind 旗下的 Isomorphic Labs 宣布，其首个完全由 AI 设计的小分子药物候选物已从 I 期推进至 II 期临床试验 —— 这是 AI 独立设计药物首次在 I 期中展现出足够的安全性和有效性信号，进入更大样本的疗效测试阶段。 AI 不再只是 "辅助工具"，它已经开始独立产出药物候选物，并走到了人体验证这一步。理性边界：不是所有罕见病都有救了 在为突破欢呼的同时，必须说清楚几个现实： 第一，这 4 款药覆盖的只是冰山一角。全球已知罕见病超过 7000 种，获批治疗的不到 5%。2026 年获批的 4 款，针对的都是机制相对清晰、技术路径成熟的单基因遗传病。更多复杂的、多基因的、尚未找到致病基因的罕见病，依然在黑暗中。 第二，基因疗法不是 "打了就好"。目前获批的基因疗法大多是 "加速批准"，意味着需要在上市后继续验证长期疗效和安全性。AAV 载体的免疫反应、基因表达的长期稳定性、潜在的插入突变风险，都需要长达数年的随访来确认。 第三，价格和可及性是巨大挑战。这些疗法定价在百万美元级别，即使有医保和支付创新，对于很多国家和患者来说仍然遥不可及。技术上的突破，不等于每个患者都能用得上。 第四，AI 加速的只是 "第一步"。截至 2026 年，直接源自 AlphaFold 结构数据的临床候选药物仍不足 12 个。药物研发是一个十步流程，AI 只加速了 "靶点结构解析" 这第一步，后续的化合物合成、动物实验、临床试验依然需要传统研发周期。AI 大幅压缩了前端时间，但尚未颠覆整个研发链条。 2026 年这 4 款罕见病基因疗法的密集获批，放在医学史上可能只是一个注脚。但对于每一个患者家庭来说，这是从 "没有希望" 到 "有药可治" 的全部距离。 那个每天凌晨 2 点起来给孩子喂玉米淀粉的妈妈，那个出生就活在无声世界里的孩子，那个反复感染、皮肤溃烂的婴儿，那个随时可能发生卒中的镰状细胞病患儿 —— 他们的人生，因为这一针而改变。 而 AI 的加入，让更多 "孤儿病" 不再是被遗忘的角落。当靶点发现的成本从几百万降到几万美元，当药物筛选的周期从几年缩短到几天，当患者招募可以通过基因组数据精准匹配 —— 罕见病药物研发的经济学，正在被彻底重写。 从无药可治到一针治愈，这条路走了几十年。但接下来的路，可能会比我们想象的更快。 本文部分内容由AI辅助生成 👉 关注我，每天拆解全球顶刊最新科学健康研究，给普通人能看懂、能落地的健康长寿指南。 免责声明：本文仅为健康生活方式科普，不构成任何医疗建议、诊断或治疗指导。如有身体不适，请及时前往正规医疗机构就诊，遵医嘱治疗。 参考信源 FDA 批准 Genglycos（Ia 型糖原贮积症）：Ultragenyx 官方新闻稿，2026-08-19 FDA 批准 Otarmeni（OTOF 遗传性耳聋）：FDA 官方公告，2026-04-23；NEJM 临床试验数据 FDA 批准 Kresladi（LAD-1）：FDA 官方公告，2026-03-26；Medscape 报道 FDA 批准 Casgevy 扩展至 2 岁以上：FDA 官方公告，2026-07-01 AI 设计药物临床管线状态报告：Boston Consulting Group 分析，Healthcare Discovery 报道，2026-05 Isomorphic Labs AI 设计药物进入 II 期临床：公司公告，2026-06 DeepMind / 华盛顿大学 AlphaFold 罕见病蛋白结构研究：The Lancet Digital Health，2026-03 全球罕见病患者数据：JMIR 2026, AI Models Could Improve Diagnosis and Care for Rare Diseases 关键词标签# 罕见病 #基因疗法 #AI 制药 #医学突破 #健康科普 #孤儿病 #CRISPR #基因治疗

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 ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Cash, cash equivalents and investments of approximately $283.7 million; expected cash runway into the second quarter of 2028 CRANBURY, N.J.--(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 Rocket previously disclosed that the initial three patients were safely dosed under the modified pivotal Phase 2 protocol for RP-A501 in Danon disease. All three patients have completed the protocol-defined safety observation period, with no thrombotic microangiopathy (TMA), capillary leak syndrome, or other significant safety concerns observed at the recalibrated dose with the refined immunomodulatory regimen. The recalibrated Phase 2 dose of 3.8 × 10¹³ GC/kg was selected based on the characteristics of the current drug product, the underlying biology of Danon disease and the Phase 1 experience, with the objective of preserving the potential for meaningful clinical benefit while optimizing RP-A501’s benefit-risk profile. Rocket is actively engaging with the FDA to seek alignment on the path to dosing additional patients and completing the pivotal Phase 2 trial and expects to provide an update on the regulatory pathway in the second half of 2026. Rocket is on track to host an investor webinar in the second half of 2026 to provide a comprehensive Danon disease program update. In parallel with the pivotal Phase 2 trial, Rocket’s global natural history study has enrolled more than 50 patients, including both males and females, building an increasingly robust longitudinal dataset to further characterize Danon disease. Continued FDA Engagement and Phase 1 Enrollment for RP-A601 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) Rocket continues to engage with the FDA regarding alignment on the design of a potential pivotal study of RP-A601 in PKP2-ACM with a regulatory update expected in the second half of 2026. Previously reported Phase 1 findings demonstrated increased PKP2 protein expression and improved desmosomal localization, together with directional improvements in arrhythmia measures and right ventricular function. 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 under NCT identifier NCT05885412. Phase 1 Trial Initiation Anticipated in 2026 for RP-A701 in BAG3-Associated Dilated Cardiomyopathy (BAG3-DCM) Patient screening and enrollment has begun, with initial patient dosing anticipated in the second half of 2026. The multicenter, dose-escalation study is 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 under NCT identifier NCT07137338. Focused Commercialization Strategy Underway for KRESLADI™ (marnetegragene autotemcel) Following FDA Approval Following the announcement of the FDA’s accelerated approval of KRESLADI™ for severe leukocyte adhesion deficiency-I (LAD-I), Rocket is implementing a focused commercialization strategy calibrated to the ultra-rare patient population and concentrated treatment landscape. The strategy is centered on a limited network of specialized Qualified Treatment Centers and an appropriately scaled commercial infrastructure, supporting patient access while maintaining disciplined investment. KRESLADI represents Rocket’s first FDA-approved product and demonstrates the Company’s ability to advance a complex genetic medicine through clinical development, CMC, regulatory review, and commercial readiness. Rocket anticipates commercial availability and the start of patient onboarding in the fourth quarter of 2026. On June 12, 2026, Rocket announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $180 million. This substantial non-dilutive capital strengthened Rocket’s financial position and supports the continued advancement of its prioritized cardiovascular pipeline. 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 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. Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Operating expenses: Research and development $ 29,497 $ 42,658 $ 60,951 $ 78,600 General and administrative 17,425 25,020 34,482 53,466 Restructuring - 3,471 - 3,471 Total operating expenses 46,922 71,149 95,433 135,537 Loss from operations (46,922 ) (71,149 ) (95,433 ) (135,537 ) Gain from sale of PRV 178,190 - 178,190 - Interest expense (473 ) (473 ) (946 ) (945 ) Interest and other income, net 347 483 508 1,819 Accretion of discount on investments, net 693 2,220 1,922 4,410 Earnings (losses) before Income Taxes 131,835 (68,919 ) 84,241 (130,253 ) Provision for Income Taxes (8,621 ) - (8,621 ) - Net income (loss) $ 123,214 $ (68,919 ) $ 75,620 $ (130,253 ) Net income (loss) per share - basic $ 1.09 $ (0.62 ) $ 0.67 $ (1.18 ) Net income (loss) per share - diluted $ 1.08 $ (0.62 ) $ 0.66 $ (1.18 ) Weighted-average common shares outstanding - basic 112,844,880 111,019,647 112,491,433 110,559,113 Weighted-average common shares outstanding - diluted 114,513,964 111,019,647 114,329,763 110,559,113 June 30, 2026 December 31, 2025 Cash, cash equivalents, and investments $ 283,747 $ 188,929 Total assets 423,098 330,449 Total liabilities 54,268 53,228 Total stockholders' equity 368,830 277,221 Contacts Investors & Media Meg Dodge mdodge@rocketpharma.com