An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene Moxeparvovec Following Plasmapheresis in Subjects With Duchenne Muscular Dystrophy and Pre-existing Antibodies to AAVrh74

This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 58 weeks.

开始日期2024-09-18

申办/合作机构

Sarepta Therapeutics, Inc.

NCT06270719

/ RecruitingN/A

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.

开始日期2024-02-07

申办/合作机构

Sarepta Therapeutics, Inc.

NCT06241950

/ Enrolling by invitation临床1期

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

开始日期2024-01-29

申办/合作机构

Sarepta Therapeutics, Inc.

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100 项与 Delandistrogene moxeparvovec 相关的临床结果

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100 项与 Delandistrogene moxeparvovec 相关的转化医学

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100 项与 Delandistrogene moxeparvovec 相关的专利（医药）

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项与 Delandistrogene moxeparvovec 相关的文献（医药）

2025-06-10·NEUROLOGY

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus

Review

作者: Dowling, James J. ; Rose, Sean C. ; Parsons, Julie A. ; Puskala Hamel, Katie ; Butterfield, Russell J. ; Silsbee, Heather M. ; Caller, Tracie Anne ; Servais, Laurent ; Bharadwaj, Jyoti ; Oskoui, Maryam ; Tolchin, Benjamin

This Evidence in Focus reviews the current evidence on the efficacy and adverse effects of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) and presents clinical considerations regarding use. The author panel systematically reviewed available clinical trial data on delandistrogene moxeparvovec in patients with DMD. The risk of bias was evaluated using the American Academy of Neurology's 2017 therapeutic classification of evidence scheme. Safety information, regulatory decisions, and clinical context were also reviewed. Six clinical trials were identified, of which 4 had peer-reviewed data available. From the 4 studies with available data (2 Class I and 2 Class III), exposure data are available on 134 boys, of which 128 are ambulatory and aged ≥4 to <8 years. Both Class I studies failed to meet the primary functional motor outcome as assessed by change in the North Star Ambulatory Assessment score. Several secondary functional motor outcomes demonstrated improvement in the treatment group with small effect sizes, not meeting statistical significance from hierarchical analysis. Corticosteroid dose exposure was higher in the treatment group in the first 12 weeks after infusion, potentially contributing to measured differences between groups. Safety outcomes were similar across studies with multiple treatment-related adverse events, including peri-infusion effects, immune myositis and myocarditis, thrombocytopenia, and liver toxicity. One death has been reported in an individual who was treated with delandistrogene moxeparvovec outside of a trial. Despite not demonstrating efficacy in its primary outcome, delandistrogene moxeparvovec has been approved by the US Food and Drug Administration (FDA) for use in boys with DMD. This decision was supported by the relative safety of the product and secondary outcome measures data in the phase 3 clinical trial. As the drug may now be actively prescribed in the United States and other countries after FDA approval, providers should be aware of the limitations of the treatment and the need to monitor for immune-related side effects including myocarditis, liver injury, and thrombocytopenia, which may require expanded clinical infrastructure. Additional clinical trials and careful collection of real-world evidence from treated patients will be essential to establish short-term and long-term effectiveness and inform understanding of benefits and risks of delandistrogene moxeparvovec across the lifespan.

2025-03-04·Expert Review of Neurotherapeutics

The latest developments in synthetic approaches to Duchenne muscular dystrophy

Review

作者: Johnson, Lucy M. ; Pulskamp, Tariq G. ; Berlau, Daniel J.

INTRODUCTION:

Duchenne muscular dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene, leading to an almost complete absence of dystrophin, which is essential for muscle cell structure and function. This resulting muscle deterioration and fibrosis, eventually causes respiratory failure and cardiomyopathy. While there is currently no cure, existing therapies aim to prolong survival and alleviate symptoms.

AREAS COVERED:

This paper reviews current and emerging therapies for DMD, focusing on their safety and efficacy. Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options. Additionally, future therapies, including gene therapy, stem cell treatments, and anti-fibrotic agents, show promise for clinical application.

EXPERT OPINION:

Advancements in DMD treatments have expanded patient options. While gene therapy offers potential for correcting the genetic defect and alleviating symptoms, corticosteroids remain the most cost-effective and well-researched treatment. This is partly due to the lack of compelling long-term safety and efficacy data for gene therapies. The accelerated FDA review process has enabled faster approval of new medications; however many have provided minimal clinical benefit to patients. Despite these challenges, continued drug development and innovative research offer hope to patients.

2025-03-01·MOLECULAR THERAPY

A comprehensive atlas of AAV tropism in the mouse

Article

作者: Cox, Aaron R ; Dickinson, Mary E ; Lutz, Cathleen M ; Lagor, William R ; Gaitan, Yaned ; Bulcha, Jote ; Hartig, Sean M ; Chuecos, Marcel A ; Suarez, Carlos Flores ; Gao, Guangping ; Hsu, Chih-Wei ; Wang, Dan ; Snow, Kathy J ; Duryea, Jeff ; Walkey, Christopher J ; Martinez, Alexa E ; Hilsenbeck, Susan G ; Hannigan, Seth ; Mirochnitchenko, Oleg ; De Giorgi, Marco ; Alves-Bezerra, Michele ; Saville, Ethan ; Ljungberg, M Cecilia ; Lanza, Denise G ; Murray, Stephen A ; Heaney, Jason D

Gene therapy with adeno-associated virus (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of 10 naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10, and AAVrh74) following systemic delivery into male and female mice. A transgene-expressing ZsGreen and Cre recombinase was used to identify transduction in a cell-dependent manner based on fluorescence. Cre-driven activation of tdTomato fluorescence offered superior sensitivity for transduced cells. All serotypes except AAV3B and AAV4 had high liver tropism. Fluorescence activation revealed transduction of unexpected tissues, including adrenals, testes, and ovaries. Rare transduced cells within tissues were also readily visualized. Biodistribution of AAV genomes correlated with fluorescence, except in immune tissues. AAV4 was found to have a pan-endothelial tropism while also targeting pancreatic beta cells. This public resource enables selection of the best AAV serotypes for basic science and preclinical applications in mice.

525

项与 Delandistrogene moxeparvovec 相关的新闻（医药）

2025-05-21

·investorrelations.sarepta.com

Sarepta Provides Update on UK Dosing in ENVISION Study of ELEVIDYS for the treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 21, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. We have received feedback from the Medicines & Healthcare products Regulatory Agency (MHRA) in the United Kingdom (U.K.) that dosing may continue uninterrupted in ENVISION, study SRP-9001-303. ENVISION is a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, and clinical trials. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20250521736806/en/ Investor Contact: Ian Estepan 617-274-4052 iestepan@sarepta.com Media Contacts: Tracy Sorrentino 617-301-8566 tsorrentino@sarepta.com Kara Hoeger 617-710-3898 KHoeger@sarepta.com Source: Sarepta Therapeutics, Inc.

临床3期基因疗法加速审批

2025-05-19

·BioSpace

ASGCT Roundup: Sarepta, Rocket, Neurogene and the World’s First Tailored CRISPR Therapy

iStock, Anton Vierietin Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized i n vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant. Over the weekend, the American Society of Gene and Cell Therapy held its annual meeting in New Orleans, hosting some of the space’s most cutting-edge research and spotlighting clinical findings that could have far-reaching implications on medical practice. BioSpace highlights some notable presentations here. A World First: Personalized CRISPR Therapy Arguably the biggest news out of ASGCT was the infant that has been treated with a “customized” base-editing therapy, which greatly eased the burden of the infant’s ultra-rare disease. The case, also published in the New England Journal of Medicine , focused on a male infant who presented with lethargy and respiratory distress within 48 hours of birth. He would eventually be diagnosed with severe carbamoyl-phosphate synthetase 1 (CSP-1) deficiency , a rare disease involving the toxic build-up of nitrogen in the body. Approximately half of babies diagnosed with the condition die “in early infancy,” according to the paper. However, after two infusions of a customized CRISPR-based gene editing therapy at 7 and 8 months of age, the patient was able to gradually increase dietary protein intake, which would otherwise have to be strongly restricted in patients with CSP-1 deficiency. Median blood ammonia levels dropped and doctors were also able to scale back nitrogen-scavenging medication. There were no severe safety concerns, according to the researchers, though they did detect “transient” elevations in liver enzymes as well as two viral infections within 4 weeks after the second dose. Rocket Shares ‘Encouraging’ Data for Cardiomyopathy Gene Therapy On Thursday, Rocket Pharmaceuticals revealed that its investigational gene therapy RP-A601 normalized right ventricle systolic function in patients with plakophilin-2 related arrhythmogenic cardiomyopathy. RP-A601 also suppressed or completely stabilized off-beat contractions of the ventricles. Aside from these clinical improvements, patients treated with RP-A601 likewise showed significantly better quality of life and functional performance. In a note to investors on Friday, BMO Capital Markets analysts called these findings “encouraging,” adding that they suggest “strong gene expression” alongside improvements in functional endpoints. On safety, the analysts noted that one patient developed a serious adverse event—Rocket did not specify what this toxicity was, revealing only that it “resolved without clinical sequelae within two months post-treatment”—though this safety signal is likely to be linked to immunomodulator use, according to BMO. While the serious safety event “can raise investor questions,” BMO argued that “the overall benefit/risk pro favorable given lack of approved disease-modifying treatments” in this space. Sarepta Provides More Cardio Color for Elevidys One of the most talked-about names at the ASGCT meeting was Elevidys, Sarepta Therapeutics ’ gene therapy for Duchenne muscular dystrophy. After all, the biologic was rocked by a patient death in March—a development that caused the biotech’s shares to crater by 22%. Sarepta sought to shore up confidence in Elevidys at ASGCT, presenting consolidated cardiovascular outcomes from four studies totaling 218 patients. Results showed that cardiac troponin levels, a marker of heart damage, fluctuated following treatment, though these were largely asymptomatic. Cardiac MRI analyses also showed that Elevidys did not result in meaningful left ventricular changes. The study also found two cases of myocarditis that arose “within days” of Elevidys treatment, though these resolved within three weeks. Overall, results showed that Elevidys had a manageable cardiac safety pro five years of follow-up. Also at ASGCT, Sarepta provided Elevidys data in ambulatory children aged 8- to 9-years—information that the space has been eagerly awaiting . These latest data show that Elevidys can elicit “ statistically significant and clinically meaningful ” functional improvements in older kids, indicating that the gene therapy can stabilize disease or slow its progression, according to Sarepta. Neurogene Unveils New Safety Protocol for Rett Syndrome Gene Therapy After Patient Death In November 2024, Neurogene reported that a young patient had died in its Phase I/II study for NGN-401, an investigational gene therapy for Rett Syndrome. A few days earlier, the patient, who received the 3E15 vg dose of NGN-401, was revealed to be in critical condition after showing signs of systemic hyperinflammation. At ASGCT, Neurogene detailed its new safety monitoring and treatment system that would allow it to detect and reverse this hyperinflammatory syndrome. In particular, the biotech conducted daily surveillance in the first week after treating patients, looking out for what it called the “three Fs:” fever, falling blood counts and elevated ferritin levels. Neurogene also implemented standard algorithms to treat systemic hyperinflammation in its trial, including high-dose corticosteroid intervention, followed by an IL-1 receptor agonist. Neurogene is moving forward with the lower, 1E15 vg dose of NGN-401, which is in Phase I/II development. The biotech “is not aware of any case of” systemic hyperinflammation at this dose level, as per a news release on Friday.

基因疗法临床结果临床研究

2025-05-17

·药明康德

首款！百时美施贵宝单抗再获欧盟委员会批准；首款杜氏肌营养不良基因疗法最新数据公布

首款！百时美施贵宝单抗再获欧盟委员会批准日前，百时美施贵宝（Bristol Myers Squibb）宣布，欧盟委员会（EC）已批准新辅助欧狄沃（Opdivo，纳武利尤单抗）联合化疗，后续进行手术及辅助欧狄沃新辅助治疗的围手术期方案，用于治疗肿瘤PD-L1表达≥1%、高风险复发的可切除非小细胞肺癌（NSCLC）成人患者。根据新闻稿，欧狄沃是在欧盟获批可同时用于可切除非小细胞肺癌围手术期治疗和仅作为新辅助治疗的首款PD-（L）1抑制剂。该决定基于CheckMate-77T研究结果，该研究评估了欧狄沃联合含铂双药化疗作为新辅助疗法，后续进行手术及欧狄沃单药辅助治疗的围手术期方案，与含铂双药化疗加安慰剂作为新辅助疗法，后续进行手术及安慰剂辅助治疗相比，用于治疗可切除NSCLC成人患者的疗效与安全性。该试验达到了无事件生存期（EFS）这一主要终点，表明在中位随访25.4个月后，与化疗加安慰剂组相比，欧狄沃组患者疾病复发、进展或死亡风险降低了42%（HR=0.58；95% CI：0.43-0.78；P=0.00025）。此外，24个月后，欧狄沃组合组患者的无事件生存率为65%，而化疗加安慰剂组为44%。该试验还在病理学完全缓解（pCR）和主要病理学缓解（MPR）等次要疗效终点上显示出有临床意义的改善。该方案在所有疗效终点均显示出获益。此外，围手术期方案的安全性与先前NSCLC研究报告一致，未发现新的安全信号。欧狄沃是一种PD-1免疫检查点抑制剂，旨在帮助恢复抗肿瘤免疫反应，利用人体自身的免疫系统来对抗癌症。首款杜氏肌营养不良基因疗法最新数据公布日前，Sarepta Therapeutics在美国基因与细胞治疗学会（ASGCT）年会上公布了EMBARK研究第二部分的新数据，这些数据进一步支持了其与罗氏（Roche）联合开发的Elevidys（delandistrogene moxeparvovec）的临床益处。根据新闻稿，Elevidys是首个获批用于杜氏肌营养不良（DMD）患者的基因疗法。在EMBARK研究第二部分的最新分析中，纳入了在第一部分接受安慰剂治疗、年龄在8至9岁（n=14）的杜氏肌营养不良症患者。在Elevidys治疗一年后，与匹配的外部对照组相比，所有关键终点的组间差异（最小二乘均值）均具有统计学意义，包括北极星步行评估（NSAA）的4.75分（P=0.0026）、从地面起身时间（TTR）的6.87秒（P=0.0010）以及10米步行/跑步（10MWR）的4.76秒（P=0.0097）。ASGCT上展示的结果来自EMBARK第二部分的持续分析，该分析将63名参与者的两年结果与未接受治疗的杜氏肌营养不良症患者的外部对照组数据进行了比较。治疗两年后的结果显示，与匹配的外部对照组相比，接受Elevidys治疗的个体在多项运动功能指标上表现更好。此外，在EMBARK研究的两年期间未观察到新的安全性信号，并且在部分患者（n=16）中，从第12周到第64周，微肌营养不良蛋白表达和肌膜定位持续存在。Elevidys是一款重组基因疗法，将表达微抗肌萎缩蛋白（micro-dystrophin）的转基因包装在AAV病毒载体中，通过单次静脉注射，使得患者肌肉生成具有部分抗肌萎缩蛋白功能的重组蛋白，可以对携带任何类型DMD致病基因变异的患者生效。罗氏在2019年与Sarepta达成28.5亿美元的研发合作，共同开发这一基因疗法。参考资料：[1] Bristol Myers Squibb Receives European Commission Approval for Perioperative Regimen of Neoadjuvant Opdivo® (nivolumab) and Chemotherapy Followed by Adjuvant Opdivo for Resectable, High-Risk Non-Small Cell Lung Cancer with PD-L1 Expression ≥1%. Retrieved May 16, 2025 from https://news.bms.com/news/details/2025/Bristol-Myers-Squibb-Receives-European-Commission-Approval-for-Perioperative-Regimen-of-Neoadjuvant-Opdivo-nivolumab-and-Chemotherapy-Followed-by-Adjuvant-Opdivo-for-Resectable-High-Risk-Non-Small-Cell-Lung-Cancer-with-PD-L1-Expression-1/default.aspx[2] Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2. Retrieved May 16, 2025 from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-presents-data-american-society-gene-cell免责声明：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：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

基因疗法临床结果上市批准临床研究

100 项与 Delandistrogene moxeparvovec 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
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适应症	国家/地区	公司	日期
杜氏肌营养不良症	美国	Sarepta Therapeutics, Inc.	2023-06-22

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05096221 (EMBARK, Company_Website) 人工标引	临床3期	杜氏肌营养不良症	14	ELEVIDYS	鏇獵顧衊衊餘糧廠範製(築顧壓鹽繭範艱範艱夢) = 蓋鹹廠壓蓋淵衊蓋衊廠齋顧範範艱鹹餘製獵積 (蓋範網壓艱齋簾簾簾淵 ) 更多	积极	2025-05-16
				external control cohort	-
NCT04626674 (SRP-9001-103 \| ENDEAVOR, Company_Website) 人工标引	临床1期	杜氏肌营养不良症	13	ELEVIDYS (cohort 6, 2 years old at time of treatment)	憲艱鹽襯憲鏇蓋蓋顧遞(蓋廠艱觸簾憲壓鏇艱鬱) = 選積淵鏇願廠繭選遞糧夢膚壓憲簾鹹積衊醖襯 (蓋願淵膚餘獵蓋範糧餘 ) 更多	积极	2025-05-16
				ELEVIDYS (cohort 4, 3 years old at time of treatment)	憲艱鹽襯憲鏇蓋蓋顧遞(蓋廠艱觸簾憲壓鏇艱鬱) = 襯顧衊餘顧範築顧糧壓夢膚壓憲簾鹹積衊醖襯 (蓋願淵膚餘獵蓋範糧餘 )
Delandistrogene Moxeparvovec Clinical Trials (ASGCT2025)	N/A	杜氏肌营养不良症 Troponin-I (TnI)	156	Delandistrogene Moxeparvovec	窪糧簾選鹹衊鬱鹹夢鬱(鏇築選繭壓鹽鬱網遞築) = At week 52, cardiac MRI performed in a subset of patients from EMBARK revealed no relevant differences in cardiac function (including LVEF), volume, or mass between patients treated with delandistrogene moxeparvovec or placebo 簾鏇艱構膚範獵願艱膚 (構顧廠鹹鬱遞製壓襯襯 ) 更多	积极	2025-05-13
NCT05096221 (EMBARK \| SRP-9001-301, PipelineReview) 人工标引	临床3期	杜氏肌营养不良症	125	ELEVIDYS+Placebo (Crossover-Treated Patients)	獵窪鑰衊餘淵夢蓋鏇遞(醖範衊遞選觸遞範餘築) = 淵簾膚製糧鏇蓋鏇製夢製鹽淵遞襯範膚蓋夢觸 (選鹹鏇製憲鏇網繭艱選 )	积极	2025-01-27
NCT05096221 (EMBARK, Literature) 人工标引	临床3期	杜氏肌营养不良症	125	delandistrogene moxeparvovec	願鏇夢艱簾鏇獵繭鏇簾(蓋遞願願製夢選簾淵壓) = 膚觸製廠觸觸醖膚蓋艱醖鬱鹽築築願膚範觸鑰 (網鏇鑰憲艱製膚鬱廠襯 ) 更多	不佳	2024-10-09
				Placebo	願鏇夢艱簾鏇獵繭鏇簾(蓋遞願願製夢選簾淵壓) = 網廠積繭壓艱糧繭壓廠醖鬱鹽築築願膚範觸鑰 (網鏇鑰憲艱製膚鬱廠襯 ) 更多
NCT05096221 (EMBARK, ASGCT2024)	临床3期	杜氏肌营养不良症	125	Delandistrogene moxeparvovec	鹽鹹艱選鬱餘憲獵積鏇(鏇窪壓艱衊獵繭遞糧窪): P-Value = 0.24 更多	积极	2024-05-07
				Delandistrogene Moxeparvovec (Placebo)
NCT04626674 (ENDEAVOR, Pubmed \| Ann Neurol) 人工标引	临床1期	杜氏肌营养不良症	20	Delandistrogene moxeparvovec	夢夢衊廠壓淵衊壓繭鏇(範構餘顧蓋憲膚獵觸繭) = 窪觸遞膚構築遞製製積淵築網鬱淵築餘壓網鹹 (鏇鑰簾簾鹹憲鹽鑰製願, 42.6) 更多	积极	2023-11-01
NCT05096221 (SRP-9001-301 \| EMBARK, Corporate Publications) 人工标引	临床3期	杜氏肌营养不良症	125	ELEVIDYS	願簾糧淵衊餘鬱餘夢襯(製窪鹹醖顧壓齋餘淵蓋) = 鹽廠膚範鬱範鏇鏇襯鹽範繭遞糧餘鹹製鏇壓顧 (餘積廠鏇膚醖夢蓋窪夢 ) 未达到	积极	2023-10-30
				placebo	願簾糧淵衊餘鬱餘夢襯(製窪鹹醖顧壓齋餘淵蓋) = 憲窪鏇壓蓋淵製鑰選淵範繭遞糧餘鹹製鏇壓顧 (餘積廠鏇膚醖夢蓋窪夢 ) 未达到
FDA 人工标引	N/A	杜氏肌营养不良症	41	ELEVIDYS	獵選繭憲鹹蓋淵範廠繭(廠醖蓋憲簾製繭窪淵夢) = Most common adverse reactions across studies (incidence ≥5%) were vomiting and nausea, liver function test increased, pyrexia, and thrombocytopenia. 選獵憲淵壓醖鬱蓋築鑰 (憲鹽襯顧築選鑰襯艱壓 ) 更多	积极	2023-06-22
				Placebo
NCT03769116 (Study 102, AAN2023)	临床2期	杜氏肌营养不良症	41	Delandistrogene moxeparvovec (SRP-9001)	簾鬱網鏇願鹽壓艱願鏇(憲窪淵淵廠構餘膚齋選) = The most common treatment-related treatment-emergent adverse events (AEs) were vomiting, decreased appetite and nausea 夢糧範觸簾顧鏇簾襯積 (夢繭鏇選遞願憲餘選選 )	积极	2023-04-25
				Placebo