Phase 3, Open-label, Single-arm Study to Evaluate Efficacy and Safety of FIX Gene Transfer With PF-06838435 (rAAV-Spark100-hFIX-R338L) in Adult Male Participants With Moderately Severe to Severe Hemophilia B (FIX:C =2%) (BeneGene-2)

This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate [ABR] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).

开始日期2019-07-29

申办/合作机构

Pfizer Inc.

NCT03307980

/ Active, not recruiting临床2期

A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF PF 06838435 AND A DOSE ESCALATION SUBSTUDY IN INDIVIDUALS WITH HEMOPHILIA B

Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up. Participants in the substudy do not need to have participated in C0371005.

开始日期2017-06-22

申办/合作机构

Pfizer Inc.

NCT02484092

/ Completed临床2期

Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B

A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.

开始日期2015-11-18

申办/合作机构

Pfizer Inc.

100 项与 Fidanacogene elaparvovec 相关的临床结果

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100 项与 Fidanacogene elaparvovec 相关的转化医学

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100 项与 Fidanacogene elaparvovec 相关的专利（医药）

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项与 Fidanacogene elaparvovec 相关的文献（医药）

2024-09-26·NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B

Article

作者: Hermans, Cédric ; Cuker, Adam ; Kavakli, Kaan ; Teitel, Jerome ; Sun, Pengling ; Oldenburg, Johannes B ; Rasko, John E J ; Rupon, Jeremy ; Biondo, Francesca ; Iorio, Alfonso ; Leavitt, Andrew D ; Cerqueira, Monica H ; Shapiro, Amy D ; Katsarou-Fasouli, Olga ; Klamroth, Robert ; Fuiman, Joanne ; Fang, Annie ; Wang, Jiaan-Der ; Frenzel, Laurent ; McKay, John ; Astermark, Jan ; Ozelo, Margareth C ; Ishiguro, Akira

BACKGROUND:

Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX-Padua), was associated with sustained factor IX activity in a phase 1-2a study.

METHODS:

We conducted a phase 3 open-label study of fidanacogene elaparvovec at a dose of 5×10¹¹ vector genome copies per kilogram of body weight. Men 18 to 65 years of age with hemophilia B and a factor IX level of 2% or less were eligible for screening if they had received at least 6 months of therapy with prophylactic factor IX concentrate. The primary end point, tested for noninferiority, was the annualized bleeding rate (treated and untreated bleeding episodes) from week 12 to month 15 after treatment with fidanacogene elaparvovec as compared with the prophylaxis lead-in period. Superiority, additional efficacy end points, and safety were also assessed.

RESULTS:

Of 316 men who underwent screening for the lead-in study, 204 (64.6%) were not eligible; 188 (59.5%) of those were ineligible owing to the presence of anti-AAV neutralizing antibodies. Of the 45 participants who received fidanacogene elaparvovec, 44 completed at least 15 months of follow-up. The annualized rate of bleeding for all bleeding episodes decreased by 71%, from 4.42 (95% confidence interval [CI], 1.80 to 7.05) at baseline to 1.28 (95% CI, 0.57 to 1.98) after gene therapy, a treatment difference of -3.15 episodes (95% CI, -5.46 to -0.83; P = 0.008). This result shows the noninferiority and superiority of fidanacogene elaparvovec to prophylaxis. At 15 months, the mean factor IX activity was 26.9% (median, 22.9%; range, 1.9 to 119.0) by one-stage SynthASil assay. A total of 28 participants (62%) received glucocorticoids for increased aminotransferase levels or decreased factor IX levels (or both) starting between 11 and 123 days. No infusion-related serious adverse events, thrombotic events, development of factor IX inhibitors, or malignant conditions were observed.

CONCLUSIONS:

Fidanacogene elaparvovec was superior to prophylaxis for the treatment of participants with hemophilia B, leading to reduced bleeding and stable factor IX expression. (Funded by Pfizer; BENEGENE-2 ClinicalTrials.gov number, NCT03861273.).

2024-04-01·Drugs

Fidanacogene Elaparvovec: First Approval

Review

作者: Dhillon, Sohita

Fidanacogene elaparvovec (^PrBEQVEZ™) is an adeno-associated viral (AAV) vector-based gene therapy developed by Spark Therapeutics (a subsidiary of Roche) and Pfizer (under a license from Spark Therapeutics) for the treatment of haemophilia B. In December 2023, fidanacogene elaparvovec received its first approval for the treatment of adults (aged ≥ 18 years) with moderately severe to severe haemophilia B (congenital factor IX deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74 (AAVRh74var). Fidanacogene elaparvovec is under regulatory review in the USA and the European Union and clinical studies are ongoing in multiple countries. This article summarizes the milestones in the development of fidanacogene elaparvovec leading to this first approval for moderately severe to severe (factor IX activity ≤ 2%) haemophilia B who are negative for neutralizing antibodies to AAVRh74var.

2021-05-01·Journal of thrombosis and haemostasis : JTH2区 · 医学

Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua

2区 · 医学

Article

作者: Robinson, Mary M ; Tiefenbacher, Stefan ; High, Katherine A ; Arruda, Valder R ; Carr, Marcus E ; Murphy, John E ; Samelson-Jones, Benjamin J ; Rupon, Jeremy ; George, Lindsey A ; Rybin, Denis

BACKGROUND:

Limited information exists regarding the factor IX (FIX) coagulant activity (FIX:C) measured by different assays following FIX-Padua gene therapy.

OBJECTIVE:

Assess for the first time FIX:C in five commonly used coagulation assays in plasma samples from hemophilia B subjects receiving FIX-Padua gene transfer.

METHODS:

FIX:C was compared between central (n = 1) and local laboratories (n = 5) in the study, and across four commonly used FIX:C one-stage assays and one FIX:C chromogenic assay. For comparison, samples of pooled congenital FIX-deficient plasma spiked with purified recombinant human FIX (rHFIX)-Padua protein or rHFIX (nonacog alfa) to obtain FIX:C concentrations from ~20% to ~40% were tested.

RESULTS:

FIX:C results at local laboratories strongly correlated with central laboratory results. However, absolute values at the central laboratory were consistently lower than those at local laboratories. Across five different FIX:C assays, a consistent pattern of FIX:C was observed for subjects receiving fidanacogene elaparvovec-expressed gene transfer. Use of Actin FSL activated partial thromboplastin time (APTT) reagent in the central laboratory resulted in lower FIX:C values compared with other APTT reagents tested. The chromogenic assay determined lower FIX:C than any of the one-stage assays. The rHFIX-Padua protein-spiked samples showed similar results. In contrast, FIX:C results for rHFIX-nonacog alfa measured within 25% of expected for all one-stage assays and below 25% in the chromogenic assay.

CONCLUSIONS:

Assay-based differences in FIX:C were observed for fidanacogene elaparvovec transgene product and rHFIX-Padua protein, suggesting the variable FIX:C determined with different assay reagents is inherent to the FIX-Padua protein and is not specific to gene therapy-derived FIX-Padua.

190

项与 Fidanacogene elaparvovec 相关的新闻（医药）

2025-03-24

·BiG生物创新社

天价基因疗法，狂热追捧中踩下急刹

作者｜与安 2025年3月，罗氏制药（Roche）宣布对旗下基因疗法子公司Spark Therapeutics启动“根本性重组”，全额减记24亿美元商誉。财务数据显示，罗氏已累计为 Spark 投入超10亿美元的重组成本，其中包括2024 年1.84亿美元的前期重组费用和2025年预计达3.4亿美元的追加支出。然而，这场持续六年的资本实验最终却惨淡收场，成为基因疗法领域最惨痛的收购案例之一。而这并非个例。过去三个月，基因治疗领域在希望与阵痛中交织前行，此前昭示熊熊野心的各家药企中，不乏惨淡收场的身影。跨国药企的管线重组、战略收缩与突破转型，再次将这一赛道的复杂性与不确定性推至台前，同时也代表着曾受资本狂热吹捧的基因疗法领域正逐步向理性发展回归转折。 01 43亿美元的深刻教训技术瓶颈下散场的资本盛宴 2019年，罗氏以43亿美元天价收购Spark Therapeutics，看中的是其全球首个获批的遗传性视网膜病变疗法Luxturna，以及血友病A基因疗法SPK-8011（dirloctocogene samoparvovec）的3期临床潜力。彼时Spark市值仅22亿美元，而罗氏以溢价125%的代价收购，创下当时基因疗法领域最大并购案，折射出行业对基因疗法商业化的狂热预期。然而截至 2024 年，Luxturna 年销售额仅 2050 万美元，同比暴跌 59%.此外，SPK-8011 项目则因安全性问题被紧急叫停，不达预期的商业表现和无法突破的技术难题，直接让这项大手笔并购案从"战略收购" 降级为 "产品交易"。迅速击穿资本泡沫的主要是三重瓶颈。首先是载体免疫性难题。Luxturna依赖的AAV2血清型载体，暴露了基因疗法最顽固的技术瓶颈之一，即预存免疫与后天免疫的双重绞杀。全球约30%-60%人群因自然感染携带AAV中和抗体，导致Luxturna治疗前必须进行抗体筛查。但Spark采用的"血清型切换"策略并未突破根本限制，数据显示超60%患者治疗后仍产生新中和抗体，直接导致疗效持久性不足。而后天免疫的影响在于，即使患者通过预存免疫筛查，治疗后仍可能因免疫系统激活产生新的抗AAV2免疫反应。其次是产能不足。Spark费城工厂病毒载体年产能仅5000剂，在适应症人群本就狭窄（全球约6000名患者）的情况下，覆盖率仍旧不足85%。患者等待期过长，市场渗透未达预期。最后则是临床表现的滑铁卢，完成收购时被列为重点的SPK-8011因安全性问题被叫停，于去年年底宣布终止研究。当病毒载体的免疫原性、生产工艺、长期安全性三大技术关卡未获突破时，资本堆砌反而加速了泡沫破裂。 02 火热势头下的行业寒冬难以走出的商业化困境与此同时，罗氏的受挫并非个例。从去年开始，基因治疗领域接连出现“利空”消息，整体成绩远低于预期，市场表现陷入疲软，多家行业明星宣布因进展不佳关停管线或业务重组。 2月21日，辉瑞宣布将停止进一步开发和商业化血友病B基因疗法Beqvez（SPK-9001）。这款药物于2024年4月刚获FDA批准，定价高达350万美元（约合人民币2537万元），在2024年全美最贵药物前十排行榜中，仅次于定价 425 万美元的 Lenmeldy，并列第二。 2024年全美最贵十款药物而Beqvez从获批上市到决定撤回，还不满一年时间。辉瑞表示，如此决定是因为“患者及医生对血友病基因疗法表现出的兴趣有限”，上市至今，尚未有患者接受过该药治疗。与此同时，辉瑞彻底清空了基因疗法管线。随着Beqvez退出，该司已没有任何基因疗法处于临床或商业化阶段。实际上，彼时将基因治疗领域列为“优先发展领域”的辉瑞，近三年来正在逐步撤离基因治疗领域。2023年7月，该司将早期研发阶段的基因疗法打包以高达10亿美元的价格卖给了阿斯利康旗下的罕见病部门Alexion；2024年6月，其针对杜氏肌营养不良症（DMD）的基因疗法fordadistrogene movaparvovec因CIFFREO Ⅲ期试验结果未达主要终点，陷入停滞；而到9月，更是撤回了市场上所有批次的镰状细胞贫血疗法Oxbryta，同时终止所有相关试验。因其临床数据表明，该药在镰状细胞患者群体中的总体效益不再足以覆盖其风险。至年底，辉瑞则终止了与Sangamo Therapeutics的全球合作及许可协议，放弃共同开发治疗A型血友病的基因疗法候选药物giroctocogene fitelparvovec（Giro-vec），尽管该药在去年完成的AFFINE III期临床试验中已取得积极结果，并已准备向FDA和EMA提交监管申请。临时放弃的主要原因大致有二，一是BioMarin同适应症的基因疗法Roctavian商业化进展不佳，市场表现惨淡，可谓现成的前车之鉴；二是辉瑞血友病新型抗体疗法Marstacimab于不久前才获批上市，作为给药便利性更加的抗体，对于基因疗法的市场空间也造成了一定冲击。总得来说，就是彼时看来，这款药物似乎难以实现大卖，而三个月后折戟的Beqvez也确实证实了这一点。而就在辉瑞宣布从基因疗法退场的前一天，曾经估值百亿的蓝鸟生物宣布卖身私募资金，一同踩下急刹。 2018年初，凭借β地中海贫血疗法Zynteglo（定价280万美元）、脑肾上腺脑白质营养不良疗法Skysona（定价300万美元）和镰状细胞病疗法Lyfgenia（定价310万美元），蓝鸟生物一时声名鹊起，股价飙升至每股2300美元，市值突破300亿美元。其“一次性治愈”叙事吸引了黑石、富达等顶级资本加持，成为首个基因疗法领域“十角兽”企业。然而，高昂定价和狭窄的患者群体让其商业化之路变得十分困难。受限于保险公司等支付方在高昂定价下的报销审批挑战，三款核心产品2023年总销售额为2910万美元，至今未实现盈利，其中，Zynteglo 2023年销售额为1670万美元，截至2024年11月累计治疗35例患者；Lyfgenia则仅完成4例患者治疗，虽与Vertex的CRISPR疗法同日获批，却因价格高出90万美元且因存在安全问题遭FDA发出黑框警告，市场份额遭到挤压。去年10月，蓝鸟宣布裁员25%并启动重组，但现金流缺口仍无法填补。最终，该司以2900万美元（不足巅峰市值0.1%）被私募基金凯雷/SK资本收购，对赌条款上限仅1亿美元，蓝鸟最终“断翅”。 03 市场洗牌中的希望与曙光大额盈利并非不可能一度受挫的基因疗法领域也并非毫无商业进展。与前文几款药物相比，Sarepta Therapeutics的Elevidys表现似乎截然不同。这是全球首款DMD基因疗法，可对携带任何类型DMD致病基因变异的患者生效，很好地填补了临床上的空白。值得注意的是，这还是和罗氏联合开发的疗法，同样高成本、高定价，售价320万美元，位列全美最贵药物第四（见图1）。2023年6月获FDA批准，8月正式上市销售，2024年便首度扭亏为盈。 2024年6月，Serepta扩大了该药的适应症，从原来的仅限于 4～5 岁具有行走能力的儿童，扩展为 4 岁及以上的儿童（无论是否有行走能力），进一步拓宽了市场覆盖面。至第三季度，Sarepta实现了净利润同比增长113%的好成绩，一举达到7619万美元。而诺华在AAV基因疗法上的投入则更是成效亮眼，针对脊髓性肌萎缩症（SMA）的Zolgensma可谓销冠，据其财报显示，2024年的年销售额仍然取得了12.14亿美元的好成绩。 Zolgensma2024年净销售额或许还有一大关键原因在于适应症的选择。反观让多位巨头或挣扎前行、或认栽止损的血友病基因疗法，市场表现不佳主要还是因为有凝血因子替代疗法和双抗药物可供患者选择。自身售价不菲，疗效也并无绝对优势，在可替代方案充足的情况下，难以打开市场的情况或许也并非难以预料。然而罗氏并未完全放弃。一方面，Spark的剩余业务将整合至集团，重组不影响Luxturna的持续供应，费城创新中心的一部分研发职能也仍然予以保留；另一方面，通过与Dyno Therapeutics达成的10亿美元合作，利用后者人工智能平台开发神经疾病新型AAV载体，罗氏将转向开发针对神经疾病的下一代基因疗法。基因疗法的故事远未终章。尽管挑战重重，部分企业仍以战略调整与技术升级寻找生机。2025年的春天，基因治疗领域仍在冰火之间跋涉。但随着临床技术的进步、监管政策的完善和支付体系的创新，当基因治疗的商业化摆脱纯资本驱动，构建起“技术-临床-支付”的完整闭环，这一领域仍然有望在迎来更为明朗开阔的前景，重唱高歌，再次高速前行。参考文献 https://www.fiercepharma.com/pharma/roche-overhauls-spark-gene-therapy-unit-recording-24b-full-impairment https://www.fiercepharma.com/pharma/pfizer-empties-gene-therapy-portfolio-discontinues-hemophilia-treatment-beqvez https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy https://www.pharmaceutical-technology.com/features/the-most-expensive-drugs-in-the-us/ 公众号文章 BioSeedin柏思荟辉瑞“泪洒”基因疗法 https://news.bioon.com/article/98498658028f.html 各家药企官网新闻动态和财报数据共建Biomedical创新生态圈！如何加入BiG会员？

基因疗法并购临床3期

2025-03-18

·FierceBiotech

Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy

Arbor Biotechnologies\' series C financing follows a $215 million series B in 2021.\n As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials.The series C was led by Arch Venture Partners and TCGX, with new investors QIA, Partners Investment, Revelation Partners and Kerna Ventures joining alongside other existing investors, according to a March 18 release.The raise will support ABO-101, a CRISPR-based gene editing therapy meant to treat primary hyperoxaluria type 1 (PH1). The FDA granted the therapy both orphan drug and rare pediatric disease designations in early February.A two-part phase 1 trial of ABO-101 is slated to start this month, according to ClinicalTrials.gov. The first part of the study will test single ascending doses of the gene therapy in adults with PH1, while the second portion will test a recommended dose in children with the disease.ABO-101 is Arbor’s first program to enter human trials.The new cash will also help other branches of Arbor’s pipeline, according to the release, including an editing program for an undisclosed rare liver disease and another program targeting amyotrophic lateral sclerosis. “Arbor is developing a differentiated portfolio with first-in-class potential to deliver on the promise of CRISPR-based genetic medicines,” Keith Crandell, partner and cofounder at Arch Venture Partners, said in the release. “Arbor has established a track record of pipeline focus, coupled with execution and capital efficiency, to yield strong preclinical data supporting its pipeline.”PH1 is caused by a malfunctioning copy of the AGXT gene, which makes an enzyme called alanine-glyoxylate aminotransferase (AGT). Though AGT is created in the liver, its absence causes the buildup of oxalate downstream in the kidneys. This buildup causes symptoms like frequent kidney stones, loss of bladder control and painful urination, and, in serious cases, it can lead to kidney failure.Arbor sprouted out of the shadows in 2018 with a $15.6 million series A and a newfound CRISPR-associated enzyme called Cas13d. This enzyme’s smaller size makes packaging it into gene therapy vectors easier.Arbor followed up its debut with much bigger money at the end of 2021, signing a deal with Vertex worth up to $1.2 billion and closing a $215 million series B.Arbor’s $73.9 million series C raise comes as the gene editing field undergoes a period of self-reflection. Pfizer recently discontinued its hemophilia gene therapy Beqvez, which carried a list price of $3.5 million per person and seemingly has not been given to any patients since its April 2024 approval. And bluebird bio, a gene therapy darling once valued at $10 billion, was sold to a pair of venture capital firms last month for a comparatively paltry $29 million.Massachusetts-based Arbor is forging ahead into the clinic without Chief Scientific Officer John Murphy, Ph.D., who plans to retire. The firm is currently searching for his replacement.

基因疗法孤儿药临床1期

2025-03-14

·BioSpace

Pfizer’s Pipeline Pruning Ends Early-Stage STING Trial

iStock, tomozina Pfizer was studying PF-07820435, an orally available agonist of the STING protein, for solid tumors. Pfizer has terminated a Phase I trial of its investigational STING therapy in advanced solid tumors, according to an update to the study’s clinicaltrials.gov page on Thursday. The move to discontinue the trial of PF-07820435 was based on “strategic business reasons,” according to the webpage. “The decision was not based on any safety and/or efficacy concerns.” BioSpace has reached out to Pfizer to confirm the discontinuation and will update this space accordingly. The Phase I trial was testing PF-07820435, with or without the PD-1 inhibitor sasanlimab, in nine patients with confirmed diagnoses of solid tumors. The main objective of the study was to establish the safety of PF-07820435 and detect early signals of efficacy, as well as determine the drug’s recommended dose for further studies. The study was initiated in February last year. STING, which stands for “stimulator interferon genes,” is a protein that plays a central role in a patient’s anti-cancer immune response by activating type I interferons and other inflammatory players. An orally available drug, PF-07820435 belongs to a class of drugs called STING agonists, which work by triggering the STING pathway and promoting immune activity against cancer cells. As of this writing, PF-07820435 remains on the pharma’s pipeline page . If Pfizer does eventually decide to completely drop PF-07820435, it will be the latest casualty in the pharma’s push to trim its pipeline and refine its business strategy. In December 2024, Pfizer turned its back on Sangamo Therapeutics and its gene therapy candidate giroctocogene fitelparvovec, which the companies were previously developing for hemophilia A. Phase III data released in July 2024 showed that 84% of patients treated with giroctocogene fitelparvovec maintained factor VIII activity above 5%, and majority achieved factor VIII levels greater than 15% of normal. Pfizer and Sangamo were supposed to Biologics License Application for giroctocogene fitelparvovec this year. The termination of the companies’ partnership will take effect on April 21, after which the pharma will transition the hemophilia A program back to Sangamo. Last month, Pfizer announced it was pulling its hemophilia B gene therapy Beqvez from markets worldwide—a decision the company pinned on “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.” Pfizer, in September 2024, likewise withdrew Oxbryta, an oral hemoglobin S polymerization inhibitor that won the FDA’s approval in 2019 for sickle cell disease. The drug has since been linked to a higher occurrence of complications and death.

临床1期基因疗法上市批准临床3期临床结果

100 项与 Fidanacogene elaparvovec 相关的药物交易

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适应症	国家/地区	公司	日期
血友病B	加拿大	Pfizer Canada ULC	2023-12-27

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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


NCT03861273 (BENEGENE-2, Literature \| Pubmed \| N Engl J Med) 人工标引	临床3期	血友病B	45	Fidanacogene elaparvovec	艱構鹽餘鹽蓋蓋築膚鑰(構顧餘積鹹襯鏇醖積齋) = 襯蓋壓選築範襯淵顧願遞顧簾鑰淵膚遞淵餘憲 (餘蓋鹹廠廠餘網製膚鬱, 0.57 ~ 1.98)	优效	2024-09-25
	临床3期	血友病B	45	Prophylactic factor IX concentrate	艱構鹽餘鹽蓋蓋築膚鑰(構顧餘積鹹襯鏇醖積齋) = 糧壓窪築鹹選築網鏇憲遞顧簾鑰淵膚遞淵餘憲 (餘蓋鹹廠廠餘網製膚鬱, 1.80 ~ 7.05)	优效	2024-09-25
NCT03861273 (BENEGENE-2, CTgov)	临床3期	血友病B	51	PF-06838435 (FIX Prophylaxis)	顧範遞鏇醖繭窪遞憲襯(構製繭範窪夢網範鏇觸) = 糧遞鏇糧夢廠淵繭襯獵製願鹹壓選觸鬱憲鹹網 (鏇壓顧糧範憲鹹窪簾齋, 鹽簾構築積遞鬱觸遞築 ~ 網壓淵壓鹹憲衊醖淵構) 更多	-	2024-03-27
NCT03861273 (BENEGENE-2, CTgov)	临床3期	血友病B	51	PF-06838435 (PF-06838435)	顧範遞鏇醖繭窪遞憲襯(構製繭範窪夢網範鏇觸) = 顧廠觸構鹽構襯獵鏇製製願鹹壓選觸鬱憲鹹網 (鏇壓顧糧範憲鹹窪簾齋, 顧繭餘築齋衊窪網製衊 ~ 鹹簾廠膚網網繭齋齋鬱) 更多	-	2024-03-27
NCT03861273 (BENEGENE-2, ASH2023)	临床3期	血友病B	45	Fidanacogene elaparvovec	衊選範鹽蓋鏇遞構鏇顧(顧鹽艱齋糧鬱鹽齋蓋糧) = 窪鬱壓觸蓋簾廠積蓋餘選簾鹽願餘簾憲窪衊製 (膚鏇憲鑰齋簾願獵憲觸 )	-	2023-12-09
NCT03861273 (BENEGENE-2, Corporate Publications) 人工标引	临床3期	血友病B	-	fidanacogene elaparvovec	憲衊選醖窪齋夢糧顧選(選鑰壓選構衊獵壓遞醖) = 積糧築築遞壓廠願膚夢壓願鬱遞構構顧窪築範 (繭餘醖獵繭艱憲壓夢獵 ) 达到更多	积极	2022-12-29
ISTH2020	临床1/2期	-	-	Fidanacogene elaparvovec	糧願構窪遞窪艱壓遞選(積範艱襯廠築積壓繭蓋) = Two patients had non-related SAEs of appendicitis and emergent lumbar discectomy. Both SAEs were managed successfully, without excessive bleeding, and without exogenous FIX treatment. 淵齋蓋簾積膚製鏇簾簾 (壓築鬱遞構襯遞窪襯鹽 )	-	2020-07-12
NCT02484092 (CTgov)	临床2期	血友病B	15	SPK-9001 (SPK-9001 (5 x 10^11 vg/kg))	憲觸鏇鬱窪積廠淵蓋顧 = 憲鏇鹹艱憲顧構構網鏇顧壓繭構壓築築齋築壓 (範鬱遞獵糧網繭醖襯鏇, 鹹願顧鹹膚鬱選壓繭襯 ~ 鬱齋壓糧鹽艱鹽鏇繭構) 更多	-	2020-05-19
NCT02484092 (CTgov)	临床2期	血友病B	15	SPK-9001 (SPK-9001 (5 x 10^11 vg/kg) IV Infusion)	糧糧襯顧顧鹹獵蓋憲鹽(衊鹹醖淵獵網願齋壓顧) = 鏇糧廠壓鹽觸憲簾鏇淵憲鏇襯壓製襯淵齋選糧 (獵鹽糧淵網製齋窪製鹽, 遞糧觸製廠淵夢壓製夢 ~ 遞製繭構襯獵願淵衊簾) 更多	-	2020-05-19
SPK-9001 (ASGCT2018)	N/A	血友病B	12	SPK-9001 (5x10^11 vg/kg)	齋餘積遞範膚醖願鬱鹽(製艱夢構簾觸餘鏇願糧) = FIX:C in the five subjects with a history of HCV and stage 1-2 liver fibrosis or with combined HIV and HCV exposure did not differ significantly from other participants 蓋鬱築積網蓋廠願願壓 (選製餘齋鬱窪襯獵膚廠 ) 更多	积极	2018-05-01
NCT02484092 (Pubmed \| Br Dent J \| N Engl J Med) 人工标引	临床2期	血友病B	10	Haemophilia B gene therapy	鏇窪觸廠鹽繭餘壓願鬱(觸積憲壓廠願襯夢艱糧) = 鑰鬱繭壓築鏇選製鏇積襯願鏇觸願窪壓廠壓齋 (選築衊糧顧構觸廠願築 )	积极	2017-12-07
SPK-9001 (ASGCT2017)	N/A	血友病B	9	SPK-9001 at a dose of 5x10^11 vg/kg	淵艱觸顧膚壓範窪襯構(製簾艱膚鏇顧膚蓋願網) = Two out of 9 infused participants observed an asymptomatic increase in hepatic transaminases resulting in a tapering course of prednisolone treatment, starting at 60 mg/day. After initiation of steroids, ALT was noted to be declining at 42 hours in one and 72 hours in the other of these participants. 糧壓鹽蓋襯艱範衊齋鏇 (積齋遞顧艱齋鏇壓糧構 ) 更多	积极	2017-05-01