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更新于：2025-09-23

Vocimagene amiretrorepvec

更新于：2025-09-23

概要

概要

基本信息

药物类型

基因疗法

别名

Flucytosine prodrug activating therapy、Retroviral replicating viral、RRV

+ [10]

靶点

CDA

作用方式

调节剂

作用机制

CDA调节剂(胞嘧啶核苷脱氨酶调节剂)

治疗领域

肿瘤神经系统疾病其他疾病

在研适应症

高级别胶质瘤复发性胶质母细胞瘤胶质母细胞瘤

非在研适应症

膀胱癌结直肠癌多形性胶质母细胞瘤+ [13]

原研机构

在研机构

非在研机构

权益机构

Forte Biosciences, Inc.

杭州索元生物医药股份有限公司

最高研发阶段临床2期

首次获批日期-

最高研发阶段(中国)-

特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)

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结构/序列

Sequence Code 540803104

关联

项与 Vocimagene amiretrorepvec 相关的临床试验

NCT06504381

/ Recruiting临床1/2期IIT

A Phase I/IIa Study to Evaluate the Efficacy of DB107-RRV (Formerly Toca511), Administered to Subjects at Time of Resection and Intravenously Thereafter, in Combination With DB107-FC (Formerly Toca FC) and Radiation Therapy or DB107-FC, Temozolomide (TMZ) and Radiation Therapy in Patients With Newly Diagnosed High Grade Glioma

This is a multicenter, open-label study of DB107-RRV (formerly Toca 511) and DB107-FC (formerly Toca FC) when administered following surgical resection in newly diagnosed High Grade Glioma (HGG) patients. The study is designed to evaluate whether treatment with DB107-RRV in combination with DB107-FC when added to standard of care provides clinical benefit to newly diagnosed HGG when compared to historical performance previously determined in well controlled clinical trials published in the peer reviewed literature. This study is going to be conducted in newly diagnosed HGG patients receiving with maximum surgical resection treatment followed by radiation and temozolomide treatment using the established Stupp Protocol for O6-methylguanine-DNA methyl-transferase (MGMT) methylated patients or radiation therapy for MGMT unmethylated patients.

开始日期2025-01-08

申办/合作机构

The University of California, San Francisco

[+2]

NCT06264388

/ Recruiting临床2期IIT

A Biomarker-Guided Phase 2 Study of DB107-RRV (Retroviral Replicating Vector) Combined With DB107-Flucytosine Extended-Release Tablets in Patients With Recurrent Glioblastoma or Anaplastic Astrocytoma

The purpose of this study is to determine if the investigational products, DB107-RRV and DB107-FC, as a combination treatment will shrink high-grade glioma (HGG) in patients with recurrent/progressive, resectable or unresectable disease and increase the time that disease is controlled.

开始日期2024-05-01

申办/合作机构

University of Miami

[+2]

NCT04105374

/ Withdrawn临床2/3期IIT

A Phase II/III Randomized, Open-Label Study of Toca 511, A Retroviral Replicating Vector, Combined With Toca FC With Temozolomide and Radiation Followed by Adjuvant Temozolomide and Toca FC Compared to Temozolomide and Radiation Followed by Adjuvant Temozolomide in Patients With Newly Diagnosed Glioblastoma

This phase II/III trial studies how well vocimagene amiretrorepvec (Toca 511) and extended release flucytosine (Toca FC) work when added to the usual treatment (temozolomide and radiation therapy) in treating patients with newly diagnosed glioblastoma. Toca 511 is a live virus that has been built to carry a gene into tumor cells. This gene carries instructions that cause the tumor cells to turn Toca FC, typically used to treat fungal infections, into a drug that may kill the tumor cells. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Giving Toca 511 and Toca FC in addition to the usual treatment (temozolomide and radiation therapy) may help shrink or stabilize cancer or extend the life of patients with newly diagnosed glioblastoma.

开始日期2020-01-31

申办/合作机构

NRG Oncology

[+1]

100 项与 Vocimagene amiretrorepvec 相关的临床结果

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100 项与 Vocimagene amiretrorepvec 相关的转化医学

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100 项与 Vocimagene amiretrorepvec 相关的专利（医药）

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268

项与 Vocimagene amiretrorepvec 相关的文献（医药）

2025-11-01·AMERICAN JOURNAL OF EMERGENCY MEDICINE

Feasibility and optimization of a second-tier prehospital critical care response for major trauma in a North American urban and suburban area: A geospatial analysis and modelling study

Article

作者: Stephenson, Rachel ; Chan, Timothy C Y ; von Vopelius-Feldt, Johannes ; Sarhangian, Vahid ; Turner, Linda ; Cheskes, Sheldon ; Nolan, Brodie ; Drennan, Ian

OBJECTIVE:

Prehospital Critical Care Response Units (CCRUs) dispatched to the scene of major traumas can deliver advanced interventions at scene but are uncommon in North America. We sought to evaluate the feasibility of CCRU response to major trauma in a North American urban-suburban region.

METHODS:

We obtained ambulance record-level data from three paramedic services in Ontario, Canada (Toronto Paramedic Service, Peel Regional Paramedic Service, and Halton Region Paramedic Service) from January 2018 to December 2022 which we aggregated into calls and applied inclusion criteria targeting major trauma. We used mathematical modelling to determine the optimal placement of CCRU bases containing an RRV or RRV/helicopter for trauma response and evaluated their expected counterfactual coverage performance using simulation. Our primary metrics were the expected number of major traumas that could have been reached by CCRUs prior to EMS departure from the scene and the resulting expected average reduction in time to accessing critical care for those patients.

RESULTS:

We found the expected counterfactual coverage of two optimally placed RRV teams to be 80 % (N = 5092) of 6391 major trauma calls included. This corresponded to an expected average reduction in time to critical care of 30 min (from 47 to 17 min). We found only marginal improvement in total calls reached by CCRUs when an RRV team was replaced with an RRV/helicopter team.

CONCLUSIONS:

Our analysis supports the feasibility of CCRU response to major trauma in a North American mixed urban-suburban region and motivates further investigation into CCRUs' clinical and cost effectiveness.

2025-10-01·JOURNAL OF CHROMATOGRAPHY A

Click chemistry-driven post-imprinting of carboxylated polymer for Selective recognition of S-rivastigmine

Article

作者: Alshammari, Ahlam F ; Alshammari, Odeh A O ; AlRashidi, Aljazi Abdullah ; Al-Otaibi, Ahmed ; Alomari, Khadra B ; Alhar, Munirah S O ; Alzahrani, Elham A ; Elsayed, Nadia H ; Monier, M

Here, in the present article, a novel molecularly imprinted polymer (MIP), S-RV-P, was prepared for the recognition and separation of the S-enantiomer of rivastigmine (S-RV) from its racemic form. Synthesis involved the formation of a poly(styrene-co-maleic anhydride) (PSM) matrix that was functionalized with 2-aminoethane-1-thiol (ATH) to introduce thiol and carboxylic acid functionalities to prepare the precursor HS-P-COOH. Post-imprinting was achieved through thiol-maleimide click crosslinking via bismaleimide (BMA) facilitated by S-RV to generate the resultant S-RV-P polymer. The kinetic analysis presented second-order kinetics, while the spontaneity and optimal crosslinking at 60 °C were confirmed by thermodynamic analysis. Thermal analysis presented improved structural stability of the crosslinked polymer. Adsorption experiments demonstrated that S-RV-P had about two times greater affinity for S-RV compared to NIP, optimal pH 6-7 for adsorption, and maximum capacity 326 mg/g. Isotherm fitting was best described by the Langmuir isotherm for monolayer adsorption. Selectivity evaluation demonstrated remarkable enantioselectivity of S-RV-P for S-RV. Gravity column separation also demonstrated high-resolution chiral discrimination with 91% ee for R-RV and 97% ee for S-RV. These findings point to the success of the imprinting process and S-RV-P's potential in drug enantio‑purification.

2025-08-01·MOLECULAR THERAPY

A phase 1B trial of vocimagene amiretrorepvec in patients with advanced solid tumors: Safety, tumor homing, and immune modulatory effects

Article

作者: Shorr, Jolene ; Ostertag, Derek ; Venkat, Shree ; Thomassen, Amber ; Rodriguez-Aguirre, Maria ; Rodrigues, Olivia Roos ; Accomando, William P ; Hogan, Daniel J ; Kwon, Deukwoo ; Yavrom, Sharon ; Merchan, Jaime R ; Donahue, Arthur ; Rodon, Jordi ; Bentley, Cornelia ; Falchook, Gerald S

Vocimagene amiretrorepvec (Toca 511) is a replicating retroviral vector (RRV) encoding cytosine deaminase that converts 5-fluorocytosine (5-FC) into 5-fluorouracil in the tumor microenvironment. This study investigated the safety and immunomodulatory effects of systemically administered Toca 511 and Toca FC in advanced cancer patients. Eligible subjects received three daily intravenous (i.v.) injections of Toca 511, followed by intratumoral (i.t.) injection of Toca 511, and a 7-day course of oral Toca FC given every 4-6 weeks. Among 21 enrolled patients, the most common treatment-related adverse events were diarrhea, nausea, vomiting, and fever. Correlative studies confirmed tumor homing after intravenous virus administration. Addition of Toca FC reduced tumor myeloid cells, Tregs, and exhausted T cells, and increased CD8 T cells. Systemically, a shift in T cells from naive to effector phenotypes, expansion of CD4 memory T cells, and increases in B cells were observed. Treatment was associated with a disease control rate of 61% and median overall survival of 9.6 months. The above results confirm feasibility of systemic administration of Toca 511 in combination with oral Toca FC, successful tumor targeting, and tumor and systemic immunomodulation. Further investigation of Toca 511/Toca FC in combination with checkpoint inhibitors and/or targeted agents is warranted.

项与 Vocimagene amiretrorepvec 相关的新闻（医药）

2025-03-29

·CPHI制药在线

科睿药业抗流感1类新药玛舒拉沙韦获批上市；默沙东近20亿美元引进恒瑞Lp(a)抑制剂 | 制药在线一周药闻复盘

点击蓝字关注我们本周，热点较多。首先看审评审批方面，国内外都有两个重磅药获批，国内来说，科睿药业抗流感1类新药玛舒拉沙韦获批上市，国外而言，first-in-class口服抗生素获FDA批准上市，成为近30年来首个获批治疗uUTI的新型口服抗生素。其次看研发方面，多个药物取得重要进展，值得一提的就是，中美瑞康公布小核酸癌症新药积极结果，完全缓解率超66%，但比较遗憾的是，Cassava Sciences阿尔茨海默病新药Ⅲ期研究失败；最后是交易及投融资方面，比较值得关注的就是，默沙东近20亿美元引进恒瑞Lp(a)抑制剂。本期盘点包括审评审批、研发以及交易及投融资三大板块，统计时间为2025.3.24-3.28，包含25条信息。审评审批NMPA上市批准1、3月27日，NMPA官网显示，青峰医药下属子公司科睿药业1类新药玛舒拉沙韦片（GP681片）获批上市，用于既往健康的12岁及以上青少年和成人单纯性甲型和乙型流感患者的治疗，不包括存在流感相关并发症高风险的患者。玛舒拉沙韦片是青峰医药与银杏树药业联合开发的一种聚合酶酸性蛋白（PA）抑制剂，患者全病程只需服药一次。申请2、3月24日，CDE官网显示，Plethora Solutions和复星医药共同申报的5.1类新药利多卡因丙胺卡因气雾剂（研发代号：PSD502）申报上市，用于治疗早泄。这是一款利多卡因及丙胺卡因的专有配方，为一款定量透皮喷雾，最早于2013年11月在欧盟获批上市，2018年12月，复星医药控股子公司万邦生化医药获得该产品在中国商业化权益。3、3月25日，CDE官网显示，施维雅申报的5.1类新药艾伏尼布片新适应症申报上市，推测适应症为联合阿扎胞甘一线治疗携带易感IDH1突变的AML患者。艾伏尼布片是一种针对IDH1突变酶的口服靶向抑制剂，此前已作为“临床急需境外新药”在国内获批，用于治疗携带IDH1易感突变的复发或难治性急性髓系白血病（AML）成人患者。临床批准 4、3月24日，CDE官网显示，恩华药业1类新药NH140068片获批临床，拟开发治疗精神分裂症。NH140068是一款适于口服的多种神经递质受体激动剂，为新一代治疗精神分裂症的创新药。本次是该产品首次在中国获批临床。5、3月25日，CDE官网显示，科伦博泰1类新药SKB107注射液获批临床，拟开发用于晚期实体瘤骨转移。这是该公司与西南医科大学附属医院共同开发的靶向肿瘤骨转移的放射性核素偶联药物（RDC）药物。本次是该产品首次在中国获批IND。6、3月25日，CDE官网显示，映恩生物1类新药注射用DB-2304获批临床，拟开发治疗系统性红斑狼疮。DB-2304是映恩生物自主研发的靶向BDCA2这一浆细胞样树突状细胞（pDC）特异靶点的ADC产品，具有潜在“first-in-class”。7、3月25日，CDE官网显示，复宏汉霖的HLX79注射液获批Ⅱ期临床，联合利妥昔单抗（汉利康）治疗活动期肾小球肾炎。HLX79是复宏汉霖许可引进的一款潜在“first-in-class”人唾液酸酶融合蛋白，2024年12月，复宏汉霖与Palleon达成一项9530万美元的授权合作，获得HLX79在中国的独家许可。8、3月27日，CDE官网显示，华东医药1类新药HDM3019获批临床，拟开发治疗类风湿关节炎。HDM3019是靶向OX40L和TNFα的双特异性抗体。华东医药于2024年8月与IMBiologics达成独家许可协议，获得两款自身免疫性疾病新药在中国等37个亚洲国家的权益，其中就包括了这款IMB-101。该项合作的总金额超3亿美元。9、3月27日，CDE官网显示，阿斯利康旗下Alexion的1类新药Eneboparatide注射液获批临床，拟开发治疗慢性甲状旁腺功能减退症。这是一款治疗性多肽，2024年3月，阿斯利康宣布以10.5亿美元收购罕见内分泌疾病领域生物技术公司Amolyt Pharma，从而获得这款名为eneboparatide（AZP-3601）的Ⅲ期临床阶段研发项目。10、3月27日，CDE官网显示，礼来申报的1类新药注射用LY4052031获批临床，拟单药用于治疗晚期或转移性尿路上皮癌或其他实体瘤。这是一款在研的抗Nectin-4抗体偶联药物（ADC），正在国际范围内开展Ⅰ期临床研究。本次是该产品首次在中国获批IND。优先审评11、3月28日，CDE官网显示，默沙东的Clesrovimab注射液拟纳入优先审评，用于即将进入或出生在第一个呼吸道合胞病毒（RSV）流行季的新生儿和婴儿预防RSV所致的下呼吸道感染。Clesrovimab是一种在研的半衰期延长的单抗，可作为被动免疫手段，用于预防RSV疾病。2024年12月，该产品向FDA批准上市，用于保护婴儿在其首个RSV季节免受RSV疾病的侵害。FDA预定在2025年6月前完成审评。突破性疗法12、3月27日，CDE官网显示，恒瑞医药1类新药HRS-5965胶囊拟纳入突破性治疗品种，适应症为原发性IgA肾病。HRS-5965为一款补体因子B抑制剂。该产品针对原发性IgA肾病适应症目前正处于Ⅱ期临床阶段。HRS-5965的首个Ⅲ期临床正在进行中，旨在评估HRS-5965胶囊对比一款C5补体抑制剂治疗阵发性睡眠性血红蛋白尿（PNH）的有效性与安全性。FDA上市批准13、3月26日，FDA官网显示，GSK的Gepotidacin（商品名：Blujepa）获批上市，用于治疗单纯性尿路感染（uUTI）的成人及12岁以上青少年患者。Gepotidacin是一款具有独特作用机制的口服“first-in-class”抗生素。14、3月26日，FDA官网显示，Exelixis的卡博替尼新适应症获批，用于治疗既往接受过治疗、不可切除的、局部晚期或转移性的、分化良好的胰腺神经内分泌肿瘤（pNET）和胰腺外NET（epNET）儿童（≥12岁）和成人患者。这是第一个获FDA批准用于系统治疗无论原发肿瘤部位、分级、生长抑素受体表达和功能状态的经治NET的药物，于2012年11月在美国获批上市。临床批准15、3月24日，FDA官网显示，神济昌华的SNUG01获批Ⅰ/Ⅱa期国际多中心注册临床试验，用于治疗肌萎缩侧索硬化（ALS）。SNUG01是以重组腺相关病毒9型（rAAV9）为载体，通过鞘内注射（IT）的方式，将人源TRIM72基因靶向递送至神经元。16、3月24日，FDA官网显示，云顶新耀的EVM14注射液获批临床。EVM14是一款靶向多种肿瘤相关抗原（TAA）的通用型的现货肿瘤治疗性疫苗，拟用于非小细胞肺癌、头颈癌等多种癌症的治疗。该产品通过将编码多种肿瘤相关抗原的mRNA原液包封在脂质系统中配制而成。优先审评17、3月25日，FDA官网显示，赛诺菲的Tolebrutinib获优先审评，用于治疗非复发性继发进展型多发性硬化症（nrSPMS）并延缓成人患者独立于复发活动的残疾进展，PDUFA日期为2025年9月28日。欧盟也正在审评该药的上市申请。如果获得批准，Tolebrutinib将成为第一种也是唯一一款既能治疗nrSPMS又能减缓残疾累积而不受复发活动影响的脑穿透性BTK抑制剂。研发临床状态18、3月24日，索元生物宣布，其DB107治疗新诊断的高级别胶质瘤的临床Ⅰ/Ⅱ期试验首例受试者完成入组。这是一项评估DB107在切除时和术后静脉注射，联合DB107FC和放射治疗或DB107FC、替莫唑胺(TMZ)和放射治疗对新诊断的高级别胶质瘤患者进行治疗的研究，计划招募70名患者，证明DB107在生物标志物阳性脑瘤患者中无进展生存率的改善。临床数据19、3月24日，Opthea Limited公布了Sozinibercept治疗湿性年龄相关性黄斑变性（wAMD）的Ⅲ期COAST研究。结果显示，该研究未达到主要终点。在总体人群中，Sozinibercept（每4周1次）联合阿柏西普组、Sozinibercept（每8周1次）联合阿柏西普组和安慰剂联合阿柏西普组的BCVA评分分别增加了13.5个字母、12.8个字母和13.7个字母，组间p值分别为0.86和0.42。Sozinibercept是一款靶向血管内皮生长因子受体3（VEGFR3）的Fc融合蛋白，可阻断VEGF-C、VEGF-D与VEGFR2、VEGFR3的结合。20、3月24日，中美瑞康公布了RAG-01治疗非肌层浸润性膀胱癌（NMIBC）Ⅰ期临床试验的积极初步数据。数据显示，在接受卡介苗（BCG）治疗失败的患者中，最低两个剂量组的原位癌（CIS）患者完全缓解率（CR）达到66.7%，且安全性表现优异。RAG-01是一种创新性的saRNA（小激活RNA）疗法，旨在上调p21肿瘤抑制基因的表达。21、3月25日，Cassava Sciences公布了Simufilam治疗轻度至中度阿尔茨海默病（AD）的Ⅲ期REFOCUS-ALZ研究数据。数据显示，未达到主要终点。此前，该研究因Ⅲ期RETHINK-ALZ研究失败在2024年11月25日终止开展。Simufilam是一款靶向错构的细丝蛋白A（FLNA）的口服小分子药物。22、3月28日，劲方医药公布了氟泽雷塞联合西妥昔单抗一线治疗NSCLC的最新Ⅱ期（KROCUS）数据。数据显示，该药具有优秀的总体疗效、显著的脑转移患者肿瘤缓解、以及优于二线及以上氟泽雷塞单药疗法的安全性/耐受性；相较于包含免疫疗法的当前一线NSCLC标准治疗，KROCUS方案也提示了针对KRAS突变患者的更优治疗潜力。氟泽雷塞为一款KRAS G12C抑制剂，由信达生物和劲方医药合作开发，是中国批准上市的首个KRAS抑制剂药物。交易及投融资23、3月24日，联邦制药宣布，其全资附属公司联邦生物已与诺和诺德签订协议。诺和诺德将获得UBT251全球（不包括中国大陆、中国香港、中国澳门和中国台湾地区）的开发、生产和商业化权益。联邦生物保留中国权益。UBT251是一款长效GLP-1R/GIPR/GCGR三重激动剂，拟开发用于治疗肥胖症、2型糖尿病和其他疾病。根据协议，联邦生物将获得2亿美元首付款和最高18亿美元的潜在里程碑付款，以及分层销售提成。24、3月25日，恒瑞医药宣布，与默沙东就其脂蛋白(a)[Lp(a)]口服小分子项目（包括名为HRS-5346先导化合物）达成独家许可协议。根据协议，恒瑞医药将HRS-5346在大中华区以外的全球范围内开发、生产和商业化的独家权利有偿许可给默沙东。恒瑞医药将收取2亿美元的首付款，并有资格获得不超过17.7亿美元的与特定的开发、监管和商业化相关的里程碑付款，以及如果相关产品获批上市，基于HRS-5346的净销售额的销售提成。25、3月26日，浦合医药宣布，与拜耳就BAY 3713372（浦合原代号PH020）达成全球许可协议。根据协议，拜耳获得开发、制造和商业化该产品的全球独家许可。BAY 3713372是一种口服的强效选择性MTA协同PRMT5抑制剂，拜耳已招募首例患者参与Ⅰ期人体首次剂量爬坡临床试验，研究该产品用于治疗MTAP缺失型实体瘤。END2025金笔奖征文活动开启，来投稿吧！领取CPHI & PMEC China 2025展会门票来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

临床3期上市批准临床申请优先审批申请上市

2025-03-26

·医药观澜

索元生物在研脑瘤基因疗法临床首位受试者完成入组

▎药明康德内容团队报道 3月24日，索元生物宣布其创新药DB107治疗新诊断的高级别胶质瘤的临床1/2期试验首例受试者完成入组。这项名为“评估DB107 RRV疗效的1/2a期研究，在切除时和术后静脉注射，联合DB107FC和放射治疗或DB107FC、替莫唑胺 (TMZ) 和放射治疗对新诊断的高级别胶质瘤患者进行治疗”的临床试验是由三家著名脑瘤研究机构，加州大学旧金山医学院、南加州大学及加州大学圣地亚哥医学院共同发起。该试验计划招募70名患者，其目标是证明DB107在生物标志物阳性脑瘤患者中无进展生存率的改善。 DB107为一种组合疗法，即由前药激活剂基因疗法的逆转录病毒复制载体（RRV） DB107-RRV（vocimagene amiretrorepvec）和口服前药DB107-FC（缓释5-氟胞嘧啶）两部分组成。DB107-RRV是一种肿瘤内和静脉注射的逆转录病毒复制载体（RRV），可将口服的5FC在肿瘤部位转化为强效化疗药5-氟尿嘧啶（5-FU）。这一疗法可使瘤内浓度达到5-FU全身用药的30-50倍，在杀死局部细胞的同时，最大限度地减少了全身暴露和5-FU的脱靶毒性。通过过往临床试验的回顾性研究，索元生物利用其独特的DGM生物标志物发现平台，发现了一个全新的生物标志物Denovo Genomic Marker 7（DGM7）。DGM7阳性的患者的总体生存率显著优于DGM7阴性患者，同时也显著优于采用标准治疗的患者。加州大学旧金山医学院神经肿瘤学家兼首席研究员Nicholas Butowski博士说：“招募第一批患者是推进高级胶质瘤患者治疗选择的重要里程碑。我们认为这项试验将为患者及提高新诊断患者的生存率提供有高度的价值。” 该项目合作单位Anova CEO Chris Beardmore博士说：“这是一项革命性的科学研究，治疗方法藉由基因疗法感染癌症细胞进行化疗。因此可以显著降低化疗治疗常见的全身毒性并且进一步感染无法通过手术和其他干预措施去除的癌细胞，大幅改善脑瘤患者的病症。” 索元生物创始人暨董事长罗文博士表示：“高级别胶质瘤（High-grade gliomas, HGG）仍然是治疗最具挑战性的癌症之一，其治疗选择有限且病情高度复杂。该疾病的治疗方法在过去20年中几乎没有变化。DGM7则是DGM发现平台首次在基因治疗药物中发现的全新生物标志物，是索元生物DGM平台在小分子药物之外的成功案例，再次证明了DGM平台的可持续性和可扩展性。这个项目将精准医疗、基因治疗及化药有机的结合起来，这种组合疗法旨在根除肿瘤细胞，同时以最小的毒性刺激强大而持久的抗癌免疫反应。” 参考资料： [1]索元生物新闻稿. 本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权及其他合作需求，请联系wuxi_media@wuxiapptec.com。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。

基因疗法放射疗法

2025-03-07

·PipelineReview

Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas

LONDON, UK & CHICAGO, IL, USA I March 05, 2025 I Anova Enterprises, Inc. (Anova), a technology-enabled CRO dedicated to accelerating promising treatments, has announced enrollment of the first two patients in the highly anticipated study of DB107 for the treatment of brain tumors. The Phase 1/2a clinical trial is a multicenter, open-label study designed to confirm whether treatment DB107, when added to standard-of-care (SOC), provides clinical benefit to patients with newly diagnosed high-grade gliomas (HGG) when compared to historical performance. The study is funded with a California Institute for Regenerative Medicine (CIRM) grant to advance the development of DB107 in patients with newly diagnosed high-grade glioma. Led by Noriyuki Kasahara, MD, PhD, Principal Investigator, Brain Tumor Research Center (BTRC) at University of California, San Francisco (UCSF), the study is also enrolling at the University of Southern California (USC) and the University of California at San Diego UCSD, and will enroll 70 participants over the coming months. “Enrolling our first patients is an important milestone in our mission to advance treatment options for patients with high- grade glioma,” said Nicholas Butowski, MD, Neuro-Oncologist and Lead Investigator at UCSF. “We are optimistic this trial will provide valuable insights into how we can better treat patients and improve survival in newly diagnosed patients.” High- grade gliomas remain one of the most challenging cancers to treat, with limited treatment options and a high degree of complexity. Treatment for the disease has not changed in 20 years. DB107 is an investigational gene therapy being developed by Denovo Biopharma for the treatment of HGG, including glioblastoma multiforme (GBM). It comprises two components: DB107-RRV (vocimagene amiretrorepvec), a retroviral replicating vector designed to selectively infect and change cancer cells, and DB107-FC, an extended-release oral formulation of 5-fluorocytosine (5-FC), which is converted into a chemotherapeutic agent within the tumor. This combination aims to eradicate tumor cells while stimulating a robust and durable anti-cancer immune response with minimal toxicity. Chris Beardmore said, “This is revolutionary science where the treatment infects cancer cells and genetically changes them to make chemotherapy inside of the infected cells. This is known to reduce systemic toxicity commonly seen with systemic treatments. It also has a chance to infect cells that cannot be removed by surgery and other interventions to improve outcomes in newly diagnosed brain tumor patients.” The clinical trial is currently recruiting eligible patients who meet the study criteria at UCSF, USC and UCSD. Individuals interested in participating or learning more about the trial can contact the study team at info.us@anovaevidence.com . To find out more, contact info.us@anovaevidence.com . About Denovo’s RRV Platform and DB107 DB107 is an innovative approach utilizing a proprietary gene therapy platform, recombinant retroviral vector (RRV) bearing cytosine deaminase, combined with a prodrug of 5-FU (5-FC), to selectively infect and kill cancer cells while stimulating a robust and durable anti-cancer immune response against a tumor with minimal toxicity. DB107 has been tested clinically in solid tumors including recurrent high-grade GBM and colorectal cancer, most recently in a randomized 403-patient Phase 3 trial. DB107 received Orphan Drug Designation in GBM from the FDA and EMA, and Fast Track Designation from the FDA. About Anova Anova Enterprises, Inc. (Anova) is technology enabled concierge research organization committed to accelerating clinical development for start-up biopharmaceutical companies utilizing the company’s proprietary technology platform (AnovaOS™). For more information, please visit www.anovaevidence.com . About Denovo Biopharma Denovo Biopharma LLC (Denovo) is a clinical‑stage biopharmaceutical company that uses a novel biomarker discovery platform including whole genome sequencing (WGS) and artificial intelligence (AI) to find new genetic biomarkers predictive of drug efficacy, and then uses these biomarkers to execute efficient clinical trials in targeted patient populations to increase the probability of success. Denovo has eight late‑stage drugs in its pipeline addressing major unmet medical needs in oncology and central nervous system diseases; most of the pipeline are first‑in‑class drugs with global rights. Denovo recently announced a major breakthrough in treatment‑resistant depression (TRD) with its DGM4™ biomarker‑guided DB104 drug (liafensine, a potential first‑in‑class triple reuptake inhibitor for TRD). The global Phase 2b clinical trial of DB104 in DGM4‑positive patients with TRD demonstrated strikingly positive results, with highly significant improvements in symptoms of depression seen in DGM4‑positive patients. Visit www.denovobiopharma.com for additional information. About the California Institute for Regenerative Medicine (CIRM) The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency’s important work. CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency’s research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state. For more information go to cirm.ca.gov . SOURCE: Anova Enterprises

临床结果临床2期孤儿药快速通道临床3期

100 项与 Vocimagene amiretrorepvec 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10328	-	-	DB15331

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
多形性胶质母细胞瘤	临床3期	美国	Tocagen, Inc.	2015-11-30
多形性胶质母细胞瘤	临床3期	加拿大	Tocagen, Inc.	2015-11-30
多形性胶质母细胞瘤	临床3期	以色列	Tocagen, Inc.	2015-11-30
复发性胶质母细胞瘤	临床3期	美国	Tocagen, Inc.	2015-11-30
复发性胶质母细胞瘤	临床3期	加拿大	Tocagen, Inc.	2015-11-30
复发性胶质母细胞瘤	临床3期	以色列	Tocagen, Inc.	2015-11-30
复发性恶性胶质瘤	临床3期	美国	Tocagen, Inc.	2015-11-30
复发性恶性胶质瘤	临床3期	加拿大	Tocagen, Inc.	2015-11-30
复发性恶性胶质瘤	临床3期	以色列	Tocagen, Inc.	2015-11-30
高级别胶质瘤	临床2期	美国	Denovo Biopharma LLC	2024-05-01

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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


NCT02414165 (TOCA 5, Pubmed \| JAMA Oncol)	临床2/3期	复发性恶性胶质瘤	403	Toca 511/FC	糧獵廠襯鏇蓋淵鬱網獵(繭糧築淵膚構選鹹淵遞) = 鬱簾蓋醖淵鑰繭鬱顧繭顧夢鏇窪簾壓淵繭積構 (鏇鑰膚糧鏇範鹹憲遞憲 )	不佳	2020-12-01
				Vocimagene Amiretrorepvec (Standard of Care (SOC))	糧獵廠襯鏇蓋淵鬱網獵(繭糧築淵膚構選鹹淵遞) = 艱憲醖餘廠膚憲選糧遞顧夢鏇窪簾壓淵繭積構 (鏇鑰膚糧鏇範鹹憲遞憲 )
NCT02576665 (Toca 6, ASCO_GI2020)	临床1期	结直肠癌	17	Toca 511 & Toca FC	積醖鹽簾觸餘獵願構糧(窪蓋蓋夢觸願襯蓋壓艱) = 夢網遞鹹鹽廠鬱範艱積餘襯製齋鑰窪窪鑰夢觸 (繭選願獵鏇選鹽構蓋構 )	积极	2020-02-04
NCT02576665 (Toca 6, SITC2019)	临床1期	实体瘤	21	Toca 511	夢遞製蓋齋繭衊衊觸夢(鹹襯觸鑰蓋襯壓願顧網) = no related Grade 4 adverse events 繭鹹觸積壓鹹餘膚遞遞 (繭窪壓願廠鹽願繭觸遞 )	积极	2019-11-01
NCT02414165 (Toca5, Biospace) 人工标引	临床3期	脑癌	-	Fluorouracil+Vocimagene amiretrorepvec	獵獵壓鹹遞願鑰製鬱範(壓繭繭衊積窪簾築構艱) = 醖衊構網廠構蓋築憲齋衊範範繭顧願襯艱鏇鑰 (襯艱艱淵遞鹹膚鹹壓鬱 )	不佳	2019-09-12
				Standard of Care	獵獵壓鹹遞願鑰製鬱範(壓繭繭衊積窪簾築構艱) = 鬱鏇鹽願鬱選壓築餘積衊範範繭顧願襯艱鏇鑰 (襯艱艱淵遞鹹膚鹹壓鬱 )
NCT01470794 (Pubmed \| Neuro Oncol) 人工标引	临床1期	复发性恶性胶质瘤	56	5-fluorocytosine+Vocimagene amiretrorepvec (IDH1 mutant and wild-type tumors)	鹹鬱憲憲廠選選膚鑰鏇(膚範鑰獵淵淵襯餘願艱) = 艱網構艱選獵淵淵淵積鏇範鹹衊蓋醖淵餘顧築 (積糧願糧襯衊築壓繭窪 ) 更多	积极	2018-09-03
NCT01470794 (ATIM-02, SNO2017)	临床1期	复发性恶性胶质瘤 IDH1 wildtype	24	Toca 511	襯窪鑰夢壓壓簾鏇製築(遞選範醖鬱壓鬱選範積) = 簾壓餘糧憲衊襯簾襯構壓齋構鹽獵餘構簾遞觸 (廠網顧選壓蓋憲繭願簾 ) 更多	积极	2017-11-06
NCT01985256 (ATIM-02, SNO2016)	临床1期	复发性恶性胶质瘤	17	Toca 511	遞簾製選廠蓋醖鹹窪遞(遞鑰齋窪顧壓製鑰壓蓋) = n=2, 18.2% 遞構繭憲壓壓憲餘願觸 (鹹積願觸蓋餘觸艱顧顧 ) 更多	积极	2016-11-07
ASTRO2016	N/A	-	-	Toca 511	遞製遞選醖襯醖繭夢襯(鹽膚網窪簾選淵廠蓋醖) = 鏇觸憲範構鑰鏇鬱窪餘糧範膚範顧製艱遞顧選 (願衊顧顧鬱糧選鹽醖夢 ) 更多	-	2016-10-01
				Toca FC	遞製遞選醖襯醖繭夢襯(鹽膚網窪簾選淵廠蓋醖) = 簾積蓋憲觸鏇觸鏇範膚糧範膚範顧製艱遞顧選 (願衊顧顧鬱糧選鹽醖夢 ) 更多
NCT02414165 (Pubmed \| Sci Transl Med) 人工标引	临床1期	复发性恶性胶质瘤	45	5-fluorocytosine+vocimagene amiretrorepvec	餘選鹽餘選壓夢壓餘壓(願鹽鬱衊選窪齋窪簾鏇) = 繭襯構獵蓋糧遞廠鏇製襯窪窪襯繭衊壓壓遞鏇 (壓蓋窪鹽夢襯襯願網繭, 10.8 ~ 20.0)	积极	2016-06-01
				5-fluorocytosine+subgroup from an external control	-
ASCO 12016 \| ASCO 22016 人工标引	临床1期	胶质母细胞瘤	-	Toca511/Toca-FC	壓獵餘餘簾夢範膚糧構(簾選壓願淵獵衊襯構艱) = 簾網鏇鏇積憲膚蓋夢顧積鹹糧遞鬱窪鹽簾繭蓋 (繭憲製鑰構廠蓋鏇廠鏇 )	积极	2016-05-20
				lomustine	壓獵餘餘簾夢範膚糧構(簾選壓願淵獵衊襯構艱) = 選積鏇窪夢淵顧範鹹鬱積鹹糧遞鬱窪鹽簾繭蓋 (繭憲製鑰構廠蓋鏇廠鏇 )