Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies

The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.

开始日期2024-01-30

申办/合作机构

CSL Behring LLC

JPRN-jRCT2033230266

/ Recruiting临床3期IIT

Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy and Safety of a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, CSL222) Administered to Japanese Adult Male Subjects with Severe or Moderately Severe Hemophilia B

开始日期2023-09-28

申办/合作机构-

NCT05962398

/ Enrolling by invitationN/A

An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B

The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).

开始日期2023-08-30

申办/合作机构

CSL Behring LLC

100 项与 Etranacogene dezaparvovec 相关的临床结果

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100 项与 Etranacogene dezaparvovec 相关的转化医学

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100 项与 Etranacogene dezaparvovec 相关的专利（医药）

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项与 Etranacogene dezaparvovec 相关的文献（医药）

2025-07-22·Blood Advances

Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years

Article

作者: Lattimore, Susan U. ; Pipe, Steven W. ; Castaman, Giancarlo ; Giermasz, Adam ; Monahan, Paul E. ; Miesbach, Wolfgang A. ; Key, Nigel S. ; von Drygalski, Annette ; Gomez, Esteban ; Leebeek, Frank W. G. ; Le Quellec, Sandra ; Recht, Michael

Abstract:

Etranacogene dezaparvovec (CSL222, formerly AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing the highly active factor IX (FIX) Padua variant controlled by a liver-specific promoter. This phase 2b, open-label, single-dose, single-arm, multicenter trial evaluated the efficacy and safety of etranacogene dezaparvovec. Three adult participants with severe or moderately severe hemophilia B (FIX ≤2%) and AAV5-neutralizing antibodies received a single IV dose (2 × 1013 genome copies per kg) of etranacogene dezaparvovec. The primary end point of FIX activity ≥5 IU/dL at 6 weeks was met (mean, 30.6 IU/dL). Secondary end points included bleed frequency, FIX concentrate use, and adverse events. Here, we report the end-of-study 5-year outcomes. After administration, mean (range) FIX activity increased to 40.8 IU/dL (31.3-50.2) at year 1 and was maintained at 45.7 IU/dL (39.0-51.2) at year 5. Mean annualized bleeding rate (all bleeds) was 0.14 for the cumulative follow-up period years 0 to 5. Two participants had 5 bleed-free years after treatment. Per protocol, 1 participant received episodic FIX replacement therapy after treatment for elective surgeries, 2 bleeding episodes, and 2 single self-administered infusions for unreported reasons. All participants discontinued and remained free of FIX prophylaxis. During the 5-year study period, there were no clinically significant elevations in liver enzymes, requirement for steroids, FIX inhibitor development, thrombotic complications, or late-emergent safety events in any participant. Five years after administration, etranacogene dezaparvovec was effective in adults with hemophilia B with a favorable safety profile. Participants are eligible to participate in an extension study (ClinicalTrials.gov identifier: NCT05962398) for 10-year additional follow-up. This trial was registered at www.clinicaltrials.gov as #NCT03489291.

2025-05-01·Research and Practice in Thrombosis and Haemostasis

Gene-based therapies for hemophilia

Article

作者: Miesbach, Wolfgang ; Glasner, Melissa F ; Pipe, Steven

Gene therapy is a transformative approach to treating genetic disorders in order to improve disease outcomes for patients. Hemophilia A and B are inherited genetic disorders caused by mutations in the FVIII and FIX genes, respectively. Traditional treatments for hemophilia have included intravenous plasma, factor concentrates, and nonfactor therapies that require lifelong prophylaxis and carry risks of factor inhibitor development. Gene therapy offers a novel solution by delivering functional FVIII or FIX genes via adeno-associated virus vectors, which enable the production of the missing factors. Clinical outcomes have shown promise through gene therapies like valoctocogene roxaparvovec for hemophilia A and etranacogene dezaparvovec and fidanacogene elaparvovec for hemophilia B. Each therapy has demonstrated efficacy in reducing bleeding rates and maintaining factor activity. However, challenges such as hepatotoxicity, immune response, and durability of gene expression persist. Future advancements aim to expand eligibility, achieve sustained expression, and minimize adverse effects. Current trials are exploring new vectors, transgenes, and methods to overcome existing limitations. Gene therapy holds the potential to revolutionize hemophilia treatment, offering a path toward long-term management and improved quality of life for patients.

2025-05-01·Applied Health Economics and Health Policy

Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA

Article

作者: Sarker, Jyotirmoy ; Tice, Jeffrey A ; Walton, Surrey M ; Rind, David M

BACKGROUND:

Hemophilia B, a severe genetic disorder, involves substantial treatment costs and frequent interventions. Etranacogene dezaparvovec (EDZ) is a recently approved gene therapy for hemophilia B.

OBJECTIVE:

This study evaluates the cost-effectiveness of EDZ compared with conventional factor IX (FIX) prophylaxis.

METHODS:

A semi-Markov model simulated a cohort of adult males with severe hemophilia B to assess the economic impact of EDZ versus FIX prophylaxis over a lifetime horizon from a health system perspective in the USA. Inputs derived from clinical trials included therapy durability and transition probabilities based on Pettersson Scores. Scenario analyses incorporated frameworks suggested by the Institute for Clinical and Economic Review for single or short-term transformative therapies.

RESULTS:

Base-case analysis showed that at a cost of US$3.5 million, EDZ led to lifetime cost savings of US$11 million and an additional 0.64 quality-adjusted life years (QALYs) compared with FIX. However, FIX has extremely high annual costs. When annual cost offsets attributed to EDZ were capped at US$150,000, EDZ was found to have a threshold price of US$3.1 million at a willingness-to-pay of US$150,000 per QALY.

CONCLUSION:

EDZ proved to be a dominant strategy over FIX prophylaxis in the base-case scenario, providing large cost savings and slightly better outcomes. The substantial costs associated with FIX are a primary driver behind these results. The introduction of cost-offset caps significantly affects the value-based price of EDZ. Using caps on cost offsets in considering price can help to balance affordability and value in the health system.

341

项与 Etranacogene dezaparvovec 相关的新闻（医药）

2025-07-23

·同写意

单次治疗近300万元，这款国内首款基因疗法贵吗？

同写意年度巨献！！本次大会特设“基因治疗”分会，7月24-26日，邀您相聚金鸡湖畔，与5000人一起为中国医药创新“同写意”！“是医保限制了我们的想象力”。7月，当我国首个获批的腺相关病毒（AAV）基因治疗药物波哌达可基注射液的价格公布时，“同写意CGT天团”微信群中，一位医药公司创始人如是调侃。2025年4月，我国生物医药领域迎来里程碑事件——信念医药研发的波哌达可基注射液（商品名：信玖凝）正式获批上市。波哌达可基注射液针对中重度血友病B成年患者群体设计。作为一种因凝血因子Ⅸ（FIX）缺乏导致的遗传性出血疾病，血友病B患者终生面临反复出血风险，关节损伤与致残率高居不下。传统治疗要求患者终身定期输注凝血因子Ⅸ，且需每周多次静脉注射，沉重的治疗负担如影随形。如今，这款承载患者希望的治疗药物价格终于得到“官宣”——单瓶定价9.3万元。根据说明书，每位患者治疗的具体用药量（即瓶数）按照患者体重计算，例如70kg体重的处方用量是44瓶。早在波哌达可基注射液获批时，“同写意CGT天团”微信群中，就有60位医药公司创始人、高管参与了“定价竞猜”。普遍的猜想价格都在100万元左右，仅有4位“群友”猜想价格达到200万元。对于新药企业，超出预计的高定价固然值得欢呼，但尚未与高定价匹配的支付模式势必也会极大地限制患者可及。TONACEA01贵吗？波哌达可基注射液采用工程化改造的嗜肝性重组腺相关病毒（rAAV）载体，通过静脉注射将优化后的人凝血因子Ⅸ基因递送至患者肝脏细胞，利用宿主细胞转录系统持续表达凝血因子Ⅸ并分泌入血，从而恢复凝血功能。Ⅲ期临床试验数据显示，输注后52周患者平均FIX活性稳定在55.08 IU/dL（正常水平50-150 IU/dL），年化出血率均值降至0.6，凝血因子输注次数从每年58.2次降至2.9次，80.8%的患者实现零出血事件。在长达4.5年的随访中，9例受试者持续维持有效凝血功能，部分患者甚至完全脱离辅助止血治疗。安全性方面，未观察到严重血栓事件或抑制物生成，主要不良事件局限于短暂转氨酶升高，经糖皮质激素干预后均可控。在定价方面，国产波哌达可基注射液采用了单瓶定价9.3万元，药量依据患者体重计算的方式。为使更多血友病B患者尽快享受到基因治疗的创新成果，北京病痛挑战公益基金会在今年发起并运行血友病慈善援助项目。针对符合申请条件且审核通过后成功入组的血友病B患者，根据医嘱进行单次治疗过程中，只需自费购买30瓶药品，高于30瓶以上的治疗药物，将由此公益项目免费援助支持。也就是说，患者自付的治疗费用最高不会超过279万元。此外，信念医药与合作伙伴武田正在积极协同多方力量探索通过多元支付、疗效保险等模式，进一步改善患者可及。信念医药董事长肖啸表示，最理想的首先是医保按疗效分期付款，其次是一次性分担现有血友病药物每年报销30万封顶的费用。他还预期，“实际上患者最后支付的价格应该会低于200万元”。TONACEA02走向“可及”虽然波哌达可基注射液与上述CAR-T疗法相比贵了两倍有余，但深入分析可见，不同疗法的价值逻辑存在本质差异。CAR-T疗法主要针对复发难治性血液肿瘤，其疗效可能随时间衰减或需二次治疗；而基因疗法追求的是单次治疗长期缓解，现有数据已证实其持续获益超过5年。从经济学视角看，按传统治疗年均40万元费用计算，7年累积支出即与基因疗法相当，而患者预期获益期可达数十年。更关键的是，基因疗法采用通用型制剂路线，相较于个体化定制的CAR-T产品，其规模化生产具备显著的降本潜力。放眼全球基因治疗市场，高价是这一领域的普遍特征。截至2025年，美国已批准超过10款基因疗法，多数定价超过千万人民币。例如Orchard公司的Lenmeldy用于治疗异染性脑白质退化症，定价高达425万美元（约3040万人民币）；CSL Behring公司的Hemgenix同样用于治疗血友病B，定价350万美元（约2500万人民币）；蓝鸟生物的Zynteglo用于β-地中海贫血，定价280万美元（约2000万人民币）。相比之下，波哌达可基注射液279万元人民币的价格仅为美国同类产品Hemgenix的约11%。需要指出的是，许多罕见病药物在中国的售价已经是全球最低。创新药研发并非计算机领域的技术迭代，Deepseek的研发成本可以降至ChatGPT的十分之一，但若把这个比例放到创新药研发上，行业则将面临巨大挑战。可喜的是，我国支付创新机制正在同步破冰。同样是在7月，国家医保局公开了基本医保目录及商保创新药目录调整工作方案和申报指南的征求意见稿。2025年基本目录调整和商保创新药目录制定同步进行，并将通过价格协商的方式，综合专家和企业的意见，将协商成功的药品纳入商保创新药目录。随着更多本土创新药企加入竞争，越来越多的创新药进入国内市场，势必会推动更多元的支付和商业化模式，基因疗法从“天价”走向“可及”的路径日渐清晰。波哌达可基注射液的上市犹如投入湖面的石子，激荡起整个产业链的涟漪。从血友病B出发，中国基因治疗产业正在完成从单点突破到生态构建的跃迁。参考文章：1.https://m.21jingji.com/article/20250719/herald/c4884dffb553a450446cfe95ae77632e.html?t=9755762.https://mp.weixin.qq.com/s/aW3Dm4ewoMm7w48vTma2NA

上市批准基因疗法临床3期

2025-06-23

·PMLiVE

First NHS patient treated with CSL Behring’s haemophilia B gene therapy Hemgenix

The first patient in the UK has been treated by the NHS with CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec). The patient from the North East of England was treated at Guys & St Thomas’s NHS Foundation Trust in London and may no longer require regular infusions to help their blood clot. Affecting more than 2,000 people in the UK, haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of clotting factor IX (FIX). Patients with severe cases of the disease currently require lifelong treatment with intravenous FIX, which can still leave them vulnerable to breakthrough bleeds and pain in the days before infusions. CSL’s Hemgenix addresses the underlying genetic cause of haemophilia B by enabling the body to continuously produce FIX, and is the only one-time gene therapy to be approved in the UK for adults with severe or moderately severe haemophilia B without a history of FIX inhibitors. Pu-Lin Luo, consultant haematologist at Guy’s and St Thomas’, said: “This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. It is also a testament to the advancement of cell and gene therapies in the UK and these are exciting times.” CSL Behring said it will continue to work “closely and collaboratively” with the selected gene therapy centres to ensure that further eligible patients across the country will be able to access Hemgenix. The National Institute for Health and Care Excellence’s decision to recommend Hemgenix for use on the NHS in June 2024 was supported by positive results from the late-stage HOPE-B trial, in which a single infusion of Hemgenix demonstrated significant increases in mean FIX activity levels of 36.9% at 18 months. These increases were sustained at 36.7% at 24 months and 38.6% at 36 months and led to an adjusted annualised bleed rate reduction of 64% at 24 and 36 months post-dose compared to the six-month lead-in period. CSL Behring also presented updated results from the ongoing trial in February this year. Data presented at this year’s Annual Congress of the European Association for Haemophilia and Allied Disorders showed that mean factor IX activity levels remained at near normal levels of 37% through four years post-treatment, and mean adjusted annualised bleeding rate for all bleeds was reduced by approximately 90% from lead-in to year four.

临床结果基因疗法上市批准

2025-06-08

·TiPLab

无效挑战者辉瑞赢得FIX AAV基因疗法的专利诉讼

2025年5月，美国联邦巡回上诉法院（CAFC）支持PTAB认定uniQure 关于FIX AAV的两项美国专利无效的决定，驳回上诉，无效挑战者辉瑞赢得了上诉。无效挑战者辉瑞赢得FIX AAV基因疗法的专利诉讼byTiPLab 木桃这一消息对于开发FIX AAV的玩家来说，无疑是个好消息，或许辉瑞帮助扫除了一个潜在的专利障碍。而且有意思的是，辉瑞已经于今年终止了B 型血友病基因疗法 Beqvez的全球商业化，而转向了治疗血友病的抗体药物。另外，该案中关于治疗方法专利的非显而易见性讨论也给了我们一些启示。血友病B是一种由于单基因突变导致的凝血因子IX（FIX）缺乏病症。传统采用高频输注重组FIX蛋白的蛋白替代疗法进行治疗，而近年来以腺相关病毒（AAV）载体为基础的基因治疗成为替代方案，将携带编码FIX蛋白的病毒载体给予患者，从而使患者自身能够持续合成FIX蛋白，降低甚至消除对蛋白替代疗法的依赖。而AAV会引发免疫反应，且达到有效治疗效果的AAV剂量可能使患者出现严重的不良免疫反应。uniQure将FIX蛋白338位的精氨酸（R）替换为亮氨酸（L）获得高活性FIX-R338L突变体，其活性比野生型FIX高8到9倍，通过降低病毒载体剂量降低患者的免疫激活。uniQure利用FIX-R338L开发了HEMGENIX®，已经获得 FDA批准。关注要点：美国专利非显而易见的判断涉案专利为US9,982,248（’248专利）和US10,465,180（’180专利），旨在通过在基因治疗中利用野生型FIX基因的突变版本来解决这一问题，分别保护涉及使用AAV递送FIX-R338L治疗凝血病和包含编码FIX-R338L的AAV载体。其中’248专利关于治疗方法类权利要求非显而易见性的观点值得关注。美国专利非显而易见的判断包括两个要点，本领域技术人员（POSA）是否有动机结合现有技术（motivation to combine）以及结合后是否有成功预期（reasonable expectation of success）。本案中，uniQure承认POSA有结合动机，主要争议点在于利用AAV-FIX-R338L基因疗法治疗血友病B是否具有合理的成功预期。’248的权利要求1： 1. A method of treating a coagulopathy in a human patient, comprising administering a vector to the human patient, wherein: a. the vector is an adeno-associated virus; b. the vector comprises a nucleic acid encoding a modified FIX polypeptide, the modified FIX polypeptide comprising at least 70% identity to SEQ ID NO: 2 and a leucine in position 338 of SEQ ID NO: 2; and c. the vector comprises promoter sequences and transcription termination and control elements; thereby treating the coagulopathy.’248 专利的独立权利要求1要求通过向人类患者施用载体来治疗凝血病，其中该载体是AAV，包含编码修饰的 FIX 多肽的核酸，其中338位是亮氨酸。PTAB认为，Stafford、Manno 和 Schuettrumpf 的组合，将使 POSA 获得合理的成功预期，即可以利用 AAV-FIX-R338L 载体治疗血友病 B。Stafford 向 POSA 证明了使用 FIX-R338A 所取得的结果也有望通过使用 FIX-R338L 实现： Stafford在实施例中明确证明了FIX-R338A的凝血活性相比于野生型提高了2-3 倍。根据权利要求，FIX-R338L 是三个明确优选的变体之一，也是单独要求保护的两个变体之一。Manno 和 Schuettrumpf 证明了 AAV 基因疗法治疗血友病 B可以成功实现： Manno 报道用 AAV递送野生型FIX 可成功实现对于血友病B的治疗； Schuettrumpf 教导了 AAV-FIX-R338A 在给小鼠注射时产生的蛋白质活性是野生型 FIX 的六倍。另外，uniQure ’248 专利描述丙氨酸（A）和亮氨酸（L）是“同源氨基酸”，并且它们之间的替换“不会改变蛋白质的表型”。CAFC观点根据CAFC最终的观点，可以概括出判断合理成功预期的两个要点1）治疗用途的合理成功预期不总是需要临床数据uniQure尝试争辩基因治疗的不可预测性，以及现有技术缺乏针对 AAV-FIX-R338L 的疗效数据。CAFC指出“在治疗方法权利要求中证明合理的成功预期不必依赖临床数据，也不必包括证明该治疗在每种情况下都会成功的确定性。“Our case law makes clear that a showing of a reasonable expectation of success in a method of treatment claim need not rely on clinical data . . . nor must it include a demonstration of certainty that the treatment would be successful in every instance.”CAFC还认为“并不总是需要体内有效性数据来证明合理的成功预期”，本案中，辉瑞的专家证人已证明“FIX 水平与临床症状有很好的相关性”，这足以构成合理的成功预期。2）保守氨基酸替换可能合理预期其效果相似性uniQure尝试争辩难以预期氨基酸替换会产生具有可预测结构和功能的突变体。CAFC认同辉瑞专家证人的观点，即 POSA应该理解丙氨酸和亮氨酸都是不带电荷、非极性、脂肪族和疏水性氨基酸，能够预期在 FIX-R338 处用亮氨酸替换丙氨酸，而不会损失凝血活性。此外，’248 专利也描述基于结构相似性，将丙氨酸和亮氨酸描述为彼此的保守替换。因此，当以多肽作为其治疗作用的分子时，如果仅是对现有蛋白质进行氨基酸保守替换在创造性上存在挑战，特别是现有技术已经披露氨基酸结构的相似性与其功能存在对应关系时。TiPLab小贴士对于治疗方法类权利要求，1）当适应症的机理比较明确时，且现有技术已经公开其类似产品的体外效果或小鼠效果，在创造性中争辩不具有“合理的成功预期”存在一定难度，比如，仅通过取得较好的临床效果争辩创造性可能还不够，可以考虑通过产品的区别特征带来的难以预期的效果和临床上其他未公开的效果，比如：更低的副作用来争辩。2）也可以通过预料不到的技术效果来争辩非显而易见，但需注意要证明其与权利要求的关联性，明确指出该预料不到的效果是由本申请的发明点带来的。在本案中uniQure指出AAV-FIX-R338L满足了长期未满足的临床需求或取得了预料不到的疗效等。CAFC认同PTAB的观点，即上述效果与权利要求缺乏足够的关联，认为该效果主要是由 Stafford 中披露的 FIX-R338L所带来的，且没有明确证明该效果由本专利保护的 AAV-FIX-R338L所带来的。3）在撰写专利文本时，描述现有技术部分需要谨慎评估对于自身创造性的影响，特别是涉及到一些已知机理描述，防止给未来授权或确权过程中埋雷。* 以上文字仅为促进讨论与交流，不构成法律意见或咨询建议。© 作为一家专业服务公司，TiPLab坚持原创的系统的研究，注重系统性知识积累与专业影响力。欢迎读者个人分享转发，各专业平台媒体如需转载请联系TiPLab获得授权。 TiPLab提供基于研究的核心知识产权服务FTO：技术商业化实施的侵权风险防范TiPLab通过对细分技术领域内核心技术和重要专利的长期研究，结合自身在数据检索和分析方面的专长，致力于帮助客户深入了解、积极应对潜在的专利侵权风险。Due Diligence：商业活动中的知识产权尽职调查在企业许可交易、融资、并购、上市等过程中，TiPLab帮助企业和投资方了解目标技术/产品的专利保护强度及产品商业化过程中潜在的专利侵权风险，从而为商业谈判和决策提供支持依据。Patenting：全球范围内高价值专利资产的创设TiPLab熟知全球主要国家和地区的法律实践和细分领域内的前沿技术进展情况，通过前瞻性的专利布局策略，帮助客户通过创设有价值的专利资产而获取、保持和巩固独特的竞争优势。

基因疗法专利无效

100 项与 Etranacogene dezaparvovec 相关的药物交易

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研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
血友病B	美国	CSL Behring LLC	2022-11-22

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临床结果

适应症

分期

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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


NCT03569891 (HOPE-B, PipelineReview) 人工标引	临床3期	血友病B	54	HEMGENIX® (etranacogene dezaparvovec-drlb)	鑰蓋夢積襯鹹艱糧廠艱(積廠簾艱觸網觸觸鬱襯) = 壓齋願襯醖餘廠廠遞窪蓋淵廠蓋遞膚繭簾窪襯 (夢壓鹽衊壓夢淵鬱衊鏇 ) 更多	积极	2025-02-07
NCT03489291 \| NCT03569891 (HOPE-B, Pubmed \| J Thromb Haemost)	临床2/3期	血友病B	29	Etranacogene dezaparvovec 2x10^13 gc/kg	築製壓範衊窪鹽餘選遞(淵願遞襯醖繭顧蓋製鏇) = 淵遞衊夢繭蓋憲簾鹹願膚鏇範顧顧鏇顧遞鏇襯 (積願鏇築繭憲選夢淵遞, 3.1 ~ 113) 更多	积极	2024-09-01
NCT03569891 (HOPE-B, NEWS) 人工标引	临床3期	血友病B	-	Hemgenix (etranacogene dezaparvovec)	廠齋壓襯餘鏇遞醖醖網(膚製壓築繭鹽鏇憲糧網) = 選範衊鑰鏇衊鹽鹹築蓋鏇醖選襯構網齋艱憲積 (積齋鏇蓋夢醖餘糧鏇願 ) 更多	积极	2024-06-28
HOPE-B Trials (ASH2023)	临床2/3期	血友病B \| HIV感染	57	Etranacogene Dezaparvovec	簾鏇淵齋遞淵餘醖窪築(壓獵憲築蓋餘夢鏇顧夢) = 壓鑰廠網壓餘艱壓網觸憲鏇選網糧襯壓簾觸觸 (觸廠衊齋蓋廠壓簾願簾 )	-	2023-12-09
NCT03569891 (HOPE-B, Pubmed \| N Engl J Med)	临床3期	血友病B	54	Etranacogene Dezaparvovec	願構觸窪醖築範餘顧餘(齋願顧選構鏇襯選衊廠) = 鹽齋壓艱糧廠簾餘壓鹽齋膚獵廠遞顧製製積鏇 (鬱膚鹹蓋糧觸製範構夢, 31.4 ~ 41.0)	积极	2023-02-23
NCT03489291 (Pubmed)	临床2期	血友病B	3	Etranacogene dezaparvovec	範願鏇願構鏇鏇願廠鹹(繭鹹築積繭遞衊網廠築) = 製遞壓廠製遞夢願範觸鑰齋糧網鏇顧憲簾窪淵 (遞製製鑰選選鑰簾齋範, 23.9 ~ 37.8)	-	2022-12-09
NCT03569891 (HOPE-B, ASH 12022 \| ASH 22022) 人工标引	临床3期	血友病B	54	Etranacogene dezaparvovec	糧夢糧壓蓋鹹願鹽簾構(壓築膚壓餘鏇選夢夢鑰) = 獵遞艱壓憲壓廠觸襯遞鏇製襯鏇積積願鹽獵窪 (繭鏇蓋憲憲夢齋獵鏇願 ) 更多	积极	2022-11-15
ESGCT2022	N/A	血友病B	-	Etranacogene dezaparvovec	觸簾壓鏇衊襯積憲鹹糧(願遞鬱築壓憲膚鹹鹹鑰) = 鑰積鹽艱鏇衊膚願選膚簾網餘鹽鏇繭壓齋築衊 (夢簾廠顧齋蓋築齋顧遞, 4.5 ~ 122.9)	-	2022-10-11
ESGCT2022	N/A	血友病B	-	Standard-of-care FIX prophylaxis	觸簾壓鏇衊襯積憲鹹糧(願遞鬱築壓憲膚鹹鹹鑰) = 壓獵淵醖鏇鏇遞鬱鏇鹹簾網餘鹽鏇繭壓齋築衊 (夢簾廠顧齋蓋築齋顧遞, 10.3 ~ 57.9)	-	2022-10-11
NCT03569891 (HOPE-B, ASGCT2022)	临床3期	血友病 AAV5 neutralizing antibodies	54	Etranacogene dezaparvovec 2x10^13 gc/kg	鏇醖夢鏇網餘窪壓簾積(廠製蓋鹽夢範鹽醖齋廠) = 願鏇選襯淵構膚襯鹹壓糧構鏇膚廠觸夢醖窪淵 (構願遞鏇築醖淵夢遞餘, 18.7)	积极	2022-05-02
NCT03569891 (ISTH2021)	临床3期	血友病B AAV5 NAbs \| FIX	54	Etranacogene Dezaparvovec	廠憲鹽膚淵遞網醖鹹醖(膚築淵襯選鬱築艱膚壓) = 蓋鬱遞繭觸壓範築鹹簾繭餘糧壓築積網衊積夢 (鹽壓遞糧齋艱構餘鬱齋 )	-	2021-07-17
NCT03569891 (ISTH2021)	临床3期	血友病B AAV5 NAbs \| FIX	54	Etranacogene Dezaparvovec	廠憲鹽膚淵遞網醖鹹醖(膚築淵襯選鬱築艱膚壓) = 遞憲觸繭鏇願網夢遞鬱繭餘糧壓築積網衊積夢 (鹽壓遞糧齋艱構餘鬱齋 )	-	2021-07-17