Fanconi Anemia

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。美国FDA批准强生与传奇生物联合开发的嵌合抗原受体（CAR）-T细胞疗法Carvykti用于二线治疗复发性或难治性多发性骨髓瘤（RRMM）成年患者。百时美施贵宝和2seventy bio 联合开发的CAR-T疗法Abecma也获得美国FDA的批准，治疗更早期的多发性骨髓瘤患者。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF———✦研发进展✦———◇美国FDA批准强生（Johnson & Johnson）与传奇生物（Legend Biotech）联合开发的嵌合抗原受体（CAR）T疗法Carvykti（ciltacabtagene autoleucel）用于治疗复发性或难治性多发性骨髓瘤（RRMM）成年患者，这些患者之前曾至少接受过包括蛋白酶体抑制剂（PI）和免疫调节剂（IMiD）的一线治疗，并对来那度胺（lenalidomide）耐药。根据新闻稿，Carvykti是首个获批用于多发性骨髓瘤患者二线或以上治疗的B细胞成熟抗原（BCMA）靶向疗法。此次批准主要基于CARTITUDE-4研究数据，这是一项国际、随机、开放标签的3期研究，用于评估Carvykti对比标准治疗方案（SOC）在既往接受过1-3线治疗的来那度胺耐药的RRMM成人患者中的疗效和安全性。研究中的SOC包括泊马度胺、硼替佐米和地塞米松（PVd）或达雷妥尤单抗、泊马度胺和地塞米松（DPd）。根据FDA所公布的ODAC会议相关文件显示，根据独立审查委员会（IRC）评估，CARTITUDE-4试验中使用Carvykti治疗患者的无进展生存期（PFS）与标准治疗组相比具有统计显著性改善（HR=0.41，95% CI：0.30-0.56，p<0.0001）。◇百时美施贵宝（Bristol Myers Squibb）和2seventy bio宣布，美国FDA基于KarMMa-3临床试验的结果，批准靶向BCMA的CAR-T细胞疗法Abecma（idecabtagene vicleucel）用于治疗复发或难治性多发性骨髓瘤成年患者，这些患者在接受包括IMiD、PI和抗CD38单克隆抗体的两种或更多先前治疗后疾病出现进展。此次批准扩大了Abecma的适应症范围，让它可以治疗更早期的多发性骨髓瘤患者。这一批准是基于3期临床试验KarMMa-3的积极结果。中位随访时间为15.9个月时，与标准方案相比，Abecma将主要终点中位PFS提高了三倍以上，达到13.3个月（95% CI：11.8-16.1），活性对照组为4.4个月（95% CI：3.4-5.9），两者具统计学差异（HR：0.49；95% CI：0.38-0.64；p<0.0001）。这表明使用Abecma治疗将疾病进展或死亡的风险降低了51%。◇Kyverna Therapeutics公司宣布，其靶向CD19的候选CAR-T疗法KYV-101在两名进展性多发性硬化（MS）患者中的研究结果在Med杂志上发表。新闻稿指出，这是KYV-101首次被用于治疗多发性硬化患者。试验结果显示，KYV-101表现出可接受的安全性特征，未发现早期神经毒性的临床征兆。在患者脑脊液中观察到CAR-T细胞的存在和扩增，并且在一名患者中观察到鞘内抗体水平的下降，这意味着CAR-T细胞可以靶向中枢神经系统中的CD19阳性B细胞。研究人员表示，这一结果支持在更为大型的临床试验中评估KYV-101。KYV-101是一款自体、全人源CD19靶向CAR-T细胞疗法，用于治疗B细胞驱动的自身免疫疾病。它目前正在1/2期临床试验中接受评估，用于治疗狼疮性肾炎患者。治疗多发性硬化和重症肌无力的IND申请也已经获得FDA的许可。◇LyGenesis公司宣布，首位终末期肝病（ESLD）患者已在该公司的2a期临床试验中接受潜在“first-in-class”再生细胞疗法的移植。这一疗法将健康供体的肝细胞移植到ESLD患者的淋巴结中，目标是在淋巴结中生长出一个或多个迷你肝脏。这些迷你肝脏能够弥补受损肝脏的功能，挽救终末期肝病患者的生命。新闻稿指出，这是首次在临床试验中利用患者自身的淋巴结作为“培养箱“来生长异位器官，代表着再生医学领域的重要里程碑。这项2a期临床试验计划招募12名ESLD患者，每位接受细胞移植的患者将接受随访1年，评估移植的同种异体肝细胞的安全性、耐受性和疗效。新闻稿指出，这一疗法可以仅用一个捐献肝脏生产的细胞，就可以治疗数十名ESLD患者，有望缓解目前移植器官短缺的问题。◇Ring Therapeutics公司宣布，该公司在预印本网站bioRxiv上发表了基于其Anellogy技术平台开发的基因疗法的临床前研究。新闻稿指出，这是首个基于人类共生病毒——指环病毒（anellovirus）的基因递送载体系统。它的独特性在于载体不会激发人体免疫反应，所以具备重复给药的潜力。这项研究描述了如何将转基因载荷装进指环病毒的衣壳，以及基于指环病毒载体的基因疗法的体外和体内实验结果。体外实验验证了基于指环病毒的载体可以转染视网膜色素上皮（RPE）细胞。在动物实验中，基于指环病毒的载体与AAV9在注射到视网膜下后达到的表达水平相似。图片来源：123RF◇Rocket Pharmaceuticals公司宣布，欧洲药品管理局（EMA）已经接受该公司为基因疗法RP-L102递交的上市申请，用于治疗特定范可尼贫血（FA）。范可尼贫血是一种罕见遗传疾病，主要特征为骨髓衰竭、癌症风险高和先天性畸形。60~70%的范可尼贫血患者携带FANCA基因突变，它编码在DNA修复中至关重要的蛋白。FANCA基因的突变导致染色体断裂和细胞对氧化和环境应激因子的敏感性。RP-L102是一款在研基因疗法，使用慢病毒载体将FANCA基因的功能性拷贝递送到患者自身的造血干细胞中。它已经获得美国FDA授予的再生医学先进疗法认定和快速通道资格，以及EMA授予的PRIME认定。◇Caribou Biosciences宣布，为同种异体CD19靶向CAR-T疗法CB-010递交的IND申请已经获得美国FDA的许可，用于治疗狼疮性肾炎（LN）和肾外狼疮（ERL）。预计CB-010治疗LN和ERL患者1期临床试验GALLOP将于2024年底前启动。CB-010是Caribou公司的同种异体CAR-T细胞疗法平台产出的主要临床阶段候选疗法。先前的报告显示，CB-010在1期临床试验ANTLER的剂量递增部分已显示出鼓舞人心的初步安全性和有效性。该试验针对的是复发或难治性B细胞非霍奇金淋巴瘤（r/r B-NHL）患者。与许多自体和异体CAR-T细胞疗法不同，CB-010的制造不依赖于慢病毒或其他逆转录病毒载体。Caribou公司的chRDNA基因编辑技术允许在T细胞DNA的预定位置精确插入表达嵌合抗原受体的转基因。———✦融资、合作、M&A✦———◇Obsidian Therapeutics宣布完成1.605亿美元C轮融资。获得资金将用于推进Obsidian主打肿瘤浸润淋巴细胞（TIL）疗法OBX-115的开发，该项目正在进行针对黑色素瘤和非小细胞肺癌患者的1/2期临床试验。OBX-115是一种新型工程化的TIL疗法，该TIL具有表达于细胞膜上受调节的白介素-15（IL-15）蛋白，因此患者在接受OBX-115治疗时不需要同时接受高剂量的白介素-2（IL-2）治疗。接受传统TIL治疗的患者需同时接受具潜在毒性的IL-2蛋白以活化体内的TIL。临床前数据显示，与未经改造的TIL疗法相比，OBX-115具有增强的持久性与效力，动物模型的肿瘤控制得到改善，因此有潜力改善不同类型实体瘤患者的临床结果。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] IECURE ANNOUNCES FDA CLEARANCE OF INVESTIGATIONAL NEW DRUG APPLICATION FOR ECUR-506 TO INITIATE OTC-HOPE TRIAL FOR TREATMENT OF NEONATAL ONSET ORNITHINE TRANSCARBAMYLASE DEFICIENCY IN THE U.S. Retrieved April 4, 2024, from https://iecure.com/news/iecure-announces-fda-clearance-of-investigational-new-drug-application-for-ecur-506-to-initiate-otc-hope-trial-for-treatment-of-neonatal-onset-ornithine-transcarbamylase-deficiency-in-the-u-s/[2] In major step toward the clinic, Ring’s human viral vector delivers gene therapy to eyes of mice. Retrieved April 4, 2024, from https://www.fiercebiotech.com/research/major-step-towards-clinic-rings-new-human-viral-vector-delivers-gene-therapy-eyes-mice[3] Myrtelle's rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from the U.S. Food and Drug Administration. Retrieved April 4, 2024, from https://www.prnewswire.com/news-releases/myrtelles-raav-olig001-aspa-gene-therapy-candidate-for-canavan-disease-receives-regenerative-medicine-advanced-therapy-rmat-designation-from-the-us-food-and-drug-administration-302104076.html[4] Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia. Retrieved April 4, 2024, from https://www.businesswire.com/news/home/20240402101364/en[5] Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease. Retrieved April 4, 2024, from https://www.globenewswire.com/news-release/2024/04/01/2855565/0/en/Ocugen-Announces-Positive-Data-and-Safety-Monitoring-Board-Review-and-Initiation-of-Enrollment-in-Medium-Dose-for-OCU410ST-a-Modifier-Gene-Therapy-in-GARDian-Study-for-Stargardt-Di.html[6] Ring Therapeutics Publishes a Novel Gene Delivery Platform Based on the Commensal Human Anellovirus. Retrieved April 4, 2024, from https://www.globenewswire.com/news-release/2024/04/01/2855145/0/en/Ring-Therapeutics-Publishes-a-Novel-Gene-Delivery-Platform-Based-on-the-Commensal-Human-Anellovirus.html[6] Obsidian Therapeutics Announces Oversubscribed $160.5 Million Series C Financing to Drive OBX-115 Clinical Development. Retrieved April 4, 2024, from https://www.businesswire.com/news/home/20240403825922/en[7] LyGenesis Announces First Patient Dosed in its Phase 2a Clinical Trial of a First-in-Class Regenerative Cell Therapy for Patients with End-Stage Liver Disease. Retrieved April 4, 2024, from https://www.prnewswire.com/news-releases/lygenesis-announces-first-patient-dosed-in-its-phase-2a-clinical-trial-of-a-first-in-class-regenerative-cell-therapy-for-patients-with-end-stage-liver-disease-302105734.html[8] First-in-Disease Use of Kyverna Therapeutics' KYV-101 in Patients With Progressive Multiple Sclerosis Published in Med. Retrieved April 4, 2024, from https://www.prnewswire.com/news-releases/first-in-disease-use-of-kyverna-therapeutics-kyv-101-in-patients-with-progressive-multiple-sclerosis-published-in-med-302103748.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

REDWOOD CITY, Calif., April 02, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CIndU), today announced that its management will present at the 23rd Annual Needham Virtual Healthcare Conference at 11am EDT on April 8, 2024. A live webcast of the presentation will be available on the Events & News – Presentations page of Jasper's Investor Relations website. An archived replay of the presentation will be available on Jasper's website for 30 days following the live broadcast. Inducement Grant On April 1, 2024, four new employees were awarded grants of options to purchase an aggregate of 22,400 shares of voting common stock (the “Options”). Each Option was granted pursuant to the Jasper Therapeutics, Inc. Amended and Restated 2022 Inducement Equity Incentive Plan, as approved by the compensation committee of Jasper’s board of directors on March 14, 2022 and as amended and restated on June 2, 2023, and was granted as an inducement material to such employee’s employment with Jasper in accordance with Nasdaq Listing Rule 5635(c)(4). The exercise price of each Option is $28.30. Each Option will vest over four years, with 25% of the total number of shares vesting on the one year anniversary of the date of commencement of such employee’s employment with Jasper and 1/48th of the total number of shares subject to each Option vesting monthly thereafter, subject in each case to such employee’s continued service to Jasper on each vesting date. Jasper is providing this information in accordance with Nasdaq Listing Rule 5635(c)(4). About Jasper Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) and as a conditioning agent for stem cell transplants for rare diseases such as sickle cell disease (SCD), Fanconi anemia (FA) and severe combined immunodeficiency (SCID). To date, briquilimab has a demonstrated efficacy and safety pro more than 145 dosed participants and healthy volunteers, with clinical outcomes as a conditioning agent in SCID, acute myeloid leukemia, MDS, FA, and SCD. For more information, please visit us at . Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding Jasper’s presentation at the 23rd Annual Needham Virtual Healthcare Conference, Jasper’s employees and equity plans and briquilimab’s potential to address mast cell driven diseases such as CSU and CIndU. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts: Joyce Allaire (investors) LifeSci Advisors 617-435-6602 jallaire@lifesciadvisors.com Alex Gray (investors) Jasper Therapeutics 650-549-1454 agray@jaspertherapeutics.com Lauren Walker (media) Real Chemistry 646-564-2156 lbarbiero@realchemistry.com

Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined with hematologic stabilization extending out to 42 months after treatment with RP-L102 CRANBURY, N.J.--(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 announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, its lentiviral (LV) vector-based investigational gene therapy for Fanconi Anemia (FA), complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder. Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma. “We are appreciative of the patients, their families, and researchers who helped reach this meaningful milestone and continue participating in the clinical development program. We look forward to partnering closely with the EMA throughout the review process to make RP-L102 available to patients with FA who are in need of new treatment options.” MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial. RP-L102 demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization. The safety pro highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated. There were no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to RP-L102. In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is BMF, which typically occurs during the first decade of life. Although allogeneic transplants can cure the hematologic component of FA, they confer significant side effects and substantially increase the risk of solid organ malignancies, which have become the most frequent cause of FA-related death. The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024. About RP-L102 RP-L102 is an investigational gene therapy that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to contain a functional copy of the FANCA gene. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. RP-L102 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. About Fanconi Anemia Fanconi Anemia (FA) is a rare genetic disorder characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is BMF, which typically occurs during the first decade of life. Allogeneic HSCT, when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Both chemotherapy conditioning and graft-versus-host disease, a known complication of allogeneic HSCT, are associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene lead to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a “gold standard” test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as “natural gene therapy” provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells. There is a high unmet medical need for patients with FA. 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 lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Our adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. 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 expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its DD program, including its planned pivotal trial, 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 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, 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, 2023, filed February 27, 2024 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: Contacts Media Kevin Giordano media@rocketpharma.com Investors Brooks Rahmer investors@rocketpharma.com Source: Rocket Pharmaceuticals, Inc. View this news release online at: