更新于：2024-05-01

GluK2

glutamate ionotropic receptor kainate type subunit 2

更新于：2024-05-01

基本信息

别名

EAA4、Excitatory amino acid receptor 4、GluK2

+ [8]

简介

Ionotropic glutamate receptor. L-glutamate acts as an excitatory neurotransmitter at many synapses in the central nervous system. Binding of the excitatory neurotransmitter L-glutamate induces a conformation change, leading to the opening of the cation channel, and thereby converts the chemical signal to an electrical impulse. The receptor then desensitizes rapidly and enters a transient inactive state, characterized by the presence of bound agonist (PubMed:28180184). Modulates cell surface expression of NETO2 (By similarity). Independent of its ionotropic glutamate receptor activity, acts as a thermoreceptor conferring sensitivity to cold temperatures (PubMed:31474366). Functions in dorsal root ganglion neurons (By similarity).

关联

项与 GluK2 相关的药物

AMT-260

靶点

GluK2 x Kainic acid receptor

作用机制

GluK2调节剂 [+2]

在研机构

uniQure NV [+1]

原研机构

uniQure biopharma BV

在研适应症

颞叶癫痫 [+1]

非在研适应症-

最高研发阶段临床1/2期

首次获批国家/地区-

首次获批日期1800-01-20

项与 GluK2 相关的临床试验

NCT06063850 / Recruiting临床1/2期

A Multi-center, Phase 1/2a, First-in-human (FIH) Study Investigating the Safety, Tolerability, and Efficacy of AMT-260 in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE) Administered Via Magnetic Resonance Imaging (MRI)-Guided Convection-enhanced Delivery (CED)

This is a study of AMT-260 in Adults with Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE). It is designed to investigate the Safety, Tolerability, and Efficacy of AMT-260 in Adults with MTLE Administered via Magnetic Resonance Imaging (MRI)-guided Convection-enhanced Delivery (CED).

开始日期2023-11-17

申办/合作机构

uniQure NV

100 项与 GluK2 相关的临床结果

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100 项与 GluK2 相关的转化医学

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0 项与 GluK2 相关的专利（医药）

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787

项与 GluK2 相关的文献（医药）

2024-02-18·FEBS letters

The positive allosteric modulator BPAM344 and L-glutamate introduce an active-like structure of the ligand-binding domain of GluK2.

Article

作者: Yasmin Bay ; Mie Egeberg Jeppesen ; Karla Frydenvang ; Pierre Francotte ; Bernard Pirotte ; Darryl S Pickering ; Anders Skov Kristensen ; Jette Sandholm Kastrup

Kainate receptors belong to the family of ionotropic glutamate receptors and contribute to the majority of fast excitatory neurotransmission. Consequently, they also play a role in brain diseases. Therefore, understanding how these receptors can be modulated is of importance. Our study provides a crystal structure of the dimeric ligand-binding domain of the kainate receptor GluK2 in complex with L-glutamate and the small-molecule positive allosteric modulator, BPAM344, in an active-like conformation. The role of Thr535 and Gln786 in modulating GluK2 by BPAM344 was investigated using a calcium-sensitive fluorescence-based assay on transiently transfected cells expressing GluK2 and mutants hereof. This study may aid in the design of compounds targeting kainate receptors, expanding their potential as targets for the treatment of brain diseases.

2024-02-08·Journal of labelled compounds & radiopharmaceuticals

Tritium and deuterium labelling of a kainate receptor antagonist and evaluation as a radioligand.

Article

作者: Paulina Chałupnik ; Aleš Marek ; Marie Emilie Leiticia Hovah ; Darryl S Pickering ; Piero Temperini ; Stephanie Donbosco ; Ewa Szymańska ; Tommy N Johansen

Kainate receptors play a crucial role in mediating synaptic transmission within the central nervous system. However, the lack of selective pharmacological tool compounds for the GluK3 subunit represents a significant challenge in studying these receptors. Recently presented compound 1 stands out as a potent antagonist of GluK3 receptors, exhibiting nanomolar affinity at GluK3 receptors and strongly inhibiting glutamate-induced currents at homomeric GluK1 and GluK3 receptors in HEK293 cells with K_b values of 65 and 39 nM, respectively. This study presents the synthesis of two potent GluK3-preferring iodine derivatives of compound 1, serving as precursors for radiolabelling. Furthermore, we demonstrate the optimisation of dehalogenation conditions using hydrogen and deuterium, resulting in [² H]-1, and demonstrate the efficient synthesis of the radioligand [³ H]-1 with a specific activity of 1.48 TBq/mmol (40.1 Ci/mmol). Radioligand binding studies conducted with [³ H]-1 as a radiotracer at GluK1, GluK2, and GluK3 receptors expressed in Sf9 and rat P2 membranes demonstrated its potential applicability for selectively studying native GluK3 receptors in the presence of GluK1 and 2-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor-blocking ligands.

2024-01-03·Hereditas

Extracellular vesicle-derived TP53BP1, CD34, and PBX1 from human peripheral blood serve as potential biomarkers for the assessment and prediction of vascular aging.

Article

作者: Yichao Wen ; Haiyang Chen ; Yu Wang ; Yiqing Sun ; Fangfang Dou ; Xiling Du ; Te Liu ; Chuan Chen

BACKGROUND:

Vascular aging is an important pathophysiological basis for the senescence of various organs and systems in the human body, and it is a common pathogenetic trigger for many chronic diseases in the elderly.

METHODS:

The extracellular vesicles (EVs) from young and aged umbilical vein endothelial cells were isolated and identified by qPCR the differential expression levels of 47 mRNAs of genes closely related to aging in the two groups.

RESULTS:

There were significant differences in the expression levels of 18 genes (we noted upregulation in PLA2G12A, TP53BP1, CD144, PDE11A, FPGT, SERPINB4, POLD1, and PPFIBP2 and downregulation in ATP2C2, ROBO2, RRM2, GUCY1B1, NAT1-14, VEGFR2, WTAPP1, CD146, DMC1, and GRIK2). Subsequent qPCR identification of the above-mentioned genes in PBMCs and plasma-EVs from the various age groups revealed that the trend in expression levels in peripheral blood plasma-EVs of the different age groups was approximately the same as that in PBMCs. Of these mRNAs, the expression of four genes-PLA2G12A, TP53BP1, OPRL1, and KIAA0895-was commensurate with increasing age. In contradistinction, the expression trend of four genes (CREG1, PBX1, CD34, and SLIT2) was inversely proportional to the increase in age. Finally, by taking their intersection, we determined that the expression of TP53BP1 was upregulated with increasing human age and that CD34 and PBX1 were downregulated with increasing age.

CONCLUSION:

Our study indicates that human peripheral blood plasma-EV-derived TP53BP1, CD34, and PBX1 potentially comprise a noninvasive biomarker for assessing and predicting vascular aging.

项与 GluK2 相关的新闻（医药）

2024-03-11

·Science Daily

Researchers uncover protein responsible for cold sensation

Researchers have identified the protein that enables mammals to sense cold, filling a long-standing knowledge gap in the field of sensory biology. University of Michigan researchers have identified the protein that enables mammals to sense cold, filling a long-standing knowledge gap in the field of sensory biology. The findings, published in Nature Neuroscience, could help unravel how we sense and suffer from cold temperature in the winter, and why some patients experience cold differently under particular disease conditions. "The field started uncovering these temperature sensors over 20 years ago, with the discovery of a heat-sensing protein called TRPV1," said neuroscientist Shawn Xu, a professor at the U-M Life Sciences Institute and a senior author of the new research. "Various studies have found the proteins that sense hot, warm, even cool temperatures -- but we've been unable to confirm what senses temperatures below about 60 degrees Fahrenheit." In a 2019 study, researchers in Xu's lab discovered the first cold-sensing receptor protein in Caenorhabditis elegans, a species of millimeter-long worms that the lab studies as a model system for understanding sensory responses. Because the gene that encodes the C. elegans protein is evolutionarily conserved across many species, including mice and humans, that finding provided a starting point for verifying the cold sensor in mammals: a protein called GluK2 (short for Glutamate ionotropic receptor kainate type subunit 2). For this latest study, a team of researchers from the Life Sciences Institute and the U-M College of Literature, Science, and the Arts tested their hypothesis in mice that were missing the GluK2 gene, and thus could not produce any GluK2 proteins. Through a series of experiments to test the animals' behavioral reactions to temperature and other mechanical stimuli, the team found that the mice responded normally to hot, warm and cool temperatures, but showed no response to noxious cold. GluK2 is primarily found on neurons in the brain, where it receives chemical signals to facilitate communication between neurons. But it is also expressed in sensory neurons in the peripheral nervous system (outside the brain and spinal cord). "We now know that this protein serves a totally different function in the peripheral nervous system, processing temperature cues instead of chemical signals to sense cold," said Bo Duan, U-M associate professor of molecular, cellular, and developmental biology and co-senior author of the study. While GluK2 is best known for its role in the brain, Xu speculates that this temperature-sensing role may have been one of the protein's original purposes. The GluK2 gene has relatives across the evolutionary tree, going all the way back to single-cell bacteria. "A bacterium has no brain, so why would it evolve a way to receive chemical signals from other neurons? But it would have great need to sense its environment, and perhaps both temperature and chemicals," said Xu, who is also a professor of molecular and integrative physiology at the U-M Medical School. "So I think temperature sensing may be an ancient function, at least for some of these glutamate receptors, that was eventually co-opted as organisms evolved more complex nervous systems." In addition to filling a gap in the temperature-sensing puzzle, Xu believes the new finding could have implications for human health and well-being. Cancer patients receiving chemotherapy, for example, often experience painful reactions to cold. "This discovery of GluK2 as a cold sensor in mammals opens new paths to better understand why humans experience painful reactions to cold, and even perhaps offers a potential therapeutic target for treating that pain in patients whose cold sensation is overstimulated," Xu said. The research was supported by the National Institutes of Health. All procedures performed in mice were approved by the Institutional Animal Care and Use Committee and performed in accordance with the institutional guidelines. Study authors are: Wei Cai, Wenwen Zhang, Chia Chun Hor, Tong Pan, Mahar Fatima, Bo Duan and X.Z. Shawn Xu of the University of Michigan; and Qin Zheng and Xinzhong Dong of the Johns Hopkins University School of Medicine

2023-10-24

·GlobeNewswire-Health Care

uniQure Announces Major Presence at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting

LEXINGTON, Mass. and AMSTERDAM, Oct. 24, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium. “Our multiple presentations at ESGCT demonstrate uniQure’s continued leadership in advancing important research within the field of gene therapy, as well as highlight our industry-leading expertise in AAV gene therapy manufacturing,” stated Richard Porter, Ph.D., Chief Business and Scientific Officer at uniQure. “We look forward to sharing these presentations that feature our technology and platform experience across numerous programs.” Specific details on uniQure’s presentations taking place at ESGCT include: Invited Talk Title: Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s DiseasePresenter: Richard Porter, Chief Business and Scientific Officer, uniQureSession Title and Location: CNS & sensory diseases II, Session 10a, Shed 2ADate and Time: Friday, October 27, 8:30 a.m. GMT+2 Title: Gene therapy for C9ORF72-ALS reduces RNA toxicity and ameliorates behavioral phenotype in ALS mouse modelPresenter: Vanessa Zancanella, Sr. Scientist Global Research, uniQureSession Title and Location: CNS & sensory diseases I, Session 2c, Maison de la PosteDate and Time: Tuesday, October 24, 17:00 – 19:00 GMT+2 Title: Pivotal safety studies for AMT-260, a novel AAV9-dual microRNA-based vector targeting GRIK2 for the treatment of temporal lobe epilepsy (Poster #433)Presenter: Nick Pearson, Sr. Director Toxicology & Translational Safety, Non-Clinical, uniQure Poster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2 Title: Production of low cross-packaging AAV capsid libraries in insect cells (Poster #166)Presenter: Bas Bosma, Scientific Leader, Technology Innovation, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15 – 19:30, Thursday October 26, 19:30 – 20:30 GMT+2 Title: Multiplexed comparison of rationally-designed AAV CNS capsids in vivo (Poster #181)Presenter: Elena Pimentel, Scientist, Technology Innovation, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2 Title: Silence and replace as potential treatment for Alzheimer Disease: in concert lowering toxic APOE and augmenting protective APOE (Poster #049)Presenter: Amila Zuko, Scientist, Global Research, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2 Title: Gene therapy for synucleinopathies: Combining vectorized antibody and miRNA-mediated lowering strategies (Poster #430)Presenter: Kim Wolzak, Jr. Scientist, Global Research, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2 Title: Alpha-synuclein lowering and rescue of motor phenotype by miRNA-based AAV gene therapy in an in vivo Parkinson’s disease rat model (Poster #420)Presenter: Rhodé van Westen Erbrink, Scientist, Global Research, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2 Title: How uniQure’s modular platform approach drives continuous innovation in AAV gene therapy development and manufacturing (Poster #329)Presenter: Erich Ehlert, Director – Process Development – Chemistry, Manufacturing and Controls, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2 Title: Accelerating process development by establishing drug substance development platform (Poster #191)Presenter: Giorgio Rainone, Bioprocess Technologist, Drug Substance Development, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2 Title: High-throughput process development in rAAV drug substance manufacturing (Poster #308)Presenter: Yang Jiang, Associate Director, Drug Substance Development, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2 Title: Platform development for AAV manufacturing with the Baculovirus Expression Vector System (BEVS) (Poster #193)Presenter: Zandra Félix Garza, Project Manager- BPU – Cells, Seeds and Research Supply, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2 About uniQure uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com uniQure Contacts: FOR INVESTORS:Chiara RussoDirect: 617-306-9137Mobile: 617-306-9137c.russo@uniQure.comFOR MEDIA:Tom MaloneDirect: 339-970-7558Mobile:339-223-8541 t.malone@uniQure.com

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·药明康德

阿尔茨海默病的鼻内疗法；潜在“first-in-class”的CDK12/13抑制剂步入临床... | 一周盘点

▎药明康德内容团队编辑本期看点1. 阿尔兹海默病（AD）鼻内疗法foralumab公布积极临床前实验结果，表明该候选疗法有可能成为治疗AD的一种无毒的新型免疫疗法。2. 靶向性放射疗法Actimab-A与挽救性化疗方案CLAG-M联用治疗复发或难治性急性髓系白血病（AML）患者的最新生存数据积极。3. 潜在“first-in-class”的CDK12/13抑制剂CT7439获批进入临床。药明康德内容团队整理Foralumab（NI-0401）：公布临床前实验结果Foralumab（之前被称为为NI-0401）是一种全人源抗CD3单克隆抗体，在健康志愿者和克罗恩病患者中静脉注射给药后，细胞因子的释放减少。在人源化小鼠模型中，口服foralumab可以调节T细胞的免疫反应并增强调节性T细胞，从而为治疗炎症和自身免疫性疾病提供益处，而通常不会发生与肠外单克隆抗体治疗相关的潜在不良事件。在此次公布结果的临床前研究中，AD模型小鼠每周接受三次鼻内foralumab治疗，为期5个月。结果显示鼻内foralumab治疗可以通过靶向激活大脑中的小胶质细胞，同时扩大外周的调节性T细胞，从而改善AD模型小鼠的病情。值得注意的是，该研究中小胶质细胞活化的减少和认知能力的改善与影响淀粉样蛋白（Aβ）的沉积无关。该研究结果表明，foralumab有可能成为治疗AD的一种无毒的新型免疫疗法。此前，美国FDA已批准foralumab的IND申请，可开展用于轻度至中度AD患者的1期临床试验。Actimab-A：公布1b期临床试验的数据Actinium Pharmaceuticals公司公布了其基于锕-225、靶向CD33的靶向性放射疗法Actimab-A与挽救性化疗方案CLAG-M联用治疗复发或难治性AML患者的1b期试验的最新生存数据。研究结果显示，所有接受了Actimab-A+CLAG-M后进行骨髓移植患者的中位总生存期（OS）为24个月。而既往接受过维奈托克（venetoclax）治疗的此类患者的中位OS为30个月。CT7439：IND申请获得FDA许可Carrick Therapeutics公司宣布美国FDA已经批准了其潜在“first-in-class”和“best-in-class”的新型细胞周期蛋白依赖性激酶12/13（CDK12/13）抑制剂CT7439的IND申请。CT7439既是CDK12/13的抑制剂，也是CDK12/13的专性辅助因子Cyclin-K的“胶水降解剂（glue degrader）”。这种双重模式大大提高了化合物的效力，并在转录水平上抑制了DNA修复。CDK12/13通过激活RNA聚合酶II来调节基因转录。在乳腺癌、卵巢癌和尤文肉瘤等多种癌症类型中，CDK12/13有望与PARP抑制剂等其他针对DNA损伤修复反应（DDR）的药物产生协同作用。该公司计划在2024年上半年启动1期临床试验，并招募包括乳腺癌、卵巢癌和尤文氏肉瘤在内的晚期实体瘤患者。PGN-EDODM1：获加拿大卫生部批准开展1期临床试验PepGen公司宣布其提交的临床试验申请（CTA）已获得加拿大卫生部的无异议函（NOL），将启动PGN-EDODM1治疗1型强直性肌营养不良症（DM1）患者的1期临床试验。此外，美国FDA已授予PGN-EDODM1孤儿药资格，用于治疗DM1。PGN-EDODM1是一种旨在恢复细胞功能的肽偶联反义寡核苷酸。DM1由CUG重复序列引起，这些重复序列在DMPK RNA中形成发夹环，导致MBNL1蛋白（一种关键的RNA处理因子蛋白）被封闭。这会导致下游错误剪接事件和许多在肌肉收缩和放松中起关键作用的蛋白质的异常表达。通过阻断有毒的CUG重复序列，PGN-EDODM1旨在恢复功能性下游剪接和肌肉功能。AMT-260：IND申请获得FDA许可AMT-260是uniQure公司开发的一种基于AAV9的一次性基因疗法，可在局部递送两种工程化的miRNA，旨在降解GRIK2基因并抑制谷氨酸受体亚型GLUK2的异常表达，拟开发治疗难治性颞叶内侧癫痫。据信，谷氨酸受体亚型GLUK2在癫痫患者的海马中异常表达时会诱发癫痫。该疗法的IND申请于近期获FDA批准。CT-0508：公布1期临床试验数据Carisma Therapeutics公司公布了其主要候选产品CT-0508的1期临床试验结果。CT-0508是一种人表皮生长因子受体2（HER2）靶向嵌合抗原受体巨噬细胞（CAR-M）疗法，用于治疗晚期/转移性HER2过度表达癌症。此次公布的结果包括第1组（n=9）和第2组（n=5）的数据。两组患者通过分次多日输注方案（第1组）或单日静脉输注（第2组）接受相同的总剂量的CT-0508。结果显示，CT-0508在两组患者中的耐受性均良好，迄今为止未报告剂量限制性毒性反应。第1组的研究结果显示，CT-0508重塑并激活了肿瘤微环境，启动了抗肿瘤T细胞免疫。第1组和第2组的转化分析结果表明，包括肿瘤微环境活化指标、T细胞活化和HER2状态在内的各种生物标志物与疾病稳定的最佳总体反应相关，为CT-0508的作用机制提供了进一步的证据。RLYB116：公布1期临床试验数据Rallybio Corporation公司公布了其用于治疗补体介导疾病的潜在长效皮下注射型C5抑制剂RLYB116在健康受试者中进行的1期临床试验数据。结果显示，在健康受试者中，单次给药100 mg和300 mg，患者的游离C5在12、24和72小时的降低率均超过99%。RLYB116的平均估计半衰期＞300小时。RLYB116单剂量给药未出现严重或重大不良事件。该候选疗法的1期多剂量递增研究正在进行中，预计将于2023年第4季度获得初步的安全性、药代动力学和药效学数据。Fostroxacitabine bralpamide（fostrox）：公布1b/2a期临床试验数据Medivir公司公布了其正在进行的1b/2a期研究的积极中期数据。该研究旨在评估fostrox联用激酶抑制剂Lenvima（lenvatinib）治疗目前一线或二线治疗无效或不能耐受的晚期肝细胞癌（HCC）患者的安全性和疗效。Fostrox是一种核苷酸DNA聚合酶抑制剂口服前药，以肝脏为靶器官，旨在选择性将药物递送到肝细胞中治疗HCC，并将副作用降至最低。此次公布的结果显示，1b期剂量递增研究的6名患者中，1名患者达到了完全缓解，2名患者达到部分缓解，2名患者达到疾病稳定。该1b阶段的中期数据进一步巩固了之前报告的安全性和有效性数据。VB.1016：2期临床的IND申请获得FDA许可VB.1016是Nykode Therapeutics公司开发的一种潜在“first-in-class”的现货型治疗性癌症候选疫苗，正在开发用于治疗人乳头瘤病毒16型（HPV16）阳性癌症。该癌症疫苗是基于Nykode的Vaccibody技术平台设计的，该平台可将抗原呈递给抗原呈递细胞。在晚期宫颈癌患者中，VB.1016联用PD-L1抑制剂atezolizumab的2期临床试验结果积极，PD-L1阳性患者的中位OS尚未达到，但分析时已超过25个月。疫苗诱导的显著HPV16特异性T细胞反应与临床反应相关。该候选药物还在一项针对癌前HPV16诱导的高级别宫颈上皮内瘤变的1/2a期研究中获得了良好的临床数据，表明免疫反应与临床反应之间存在统计学意义上的显著相关性。Nykod公司有望在2023年第4季度启动1项2期临床试验，该试验旨在评估VB.1016与atezolizumab在HPV16阳性、PD-L1阳性、复发性或转移性宫颈癌患者中的联合治疗效果。BioLexa：公布1b期临床试验数据Hoth Therapeutics公司公布了其专有的局部抗菌配方BioLexa治疗特应性皮炎的1b期临床试验结果。BioLexa的配方经过优化，可防止葡萄球菌生物膜的形成，使细菌处于更易受抗菌治疗影响的状态。这种新的作用机制有望广泛治疗葡萄球菌生物膜形成引起的临床表现。此次公布的结果显示，78%的患者表示，自从开始使用BioLexa后，他们的总体健康状况有所改善。患者报告的皮肤科生活质量指数调查表（DLQI）显示，使用BioLexa能使患者的生活质量提高46%，而安慰剂则没有任何效果。大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] Rallybio Presents Phase 1 Single Ascending Dose Data for RLYB116, an Innovative Subcutaneously Injected Inhibitor of Complement Component 5. Retrieved September 4，2023，from https://www.businesswire.com/news/home/20230828368884/en[2] Carisma Announces Latest Data from Phase 1 Clinical Trial of CT-0508 at 8th Annual CAR-TCR Summit. Retrieved September 4，2023，from https://www.prnewswire.com/news-releases/carisma-announces-latest-data-from-phase-1-clinical-trial-of-ct-0508-at-8th-annual-car-tcr-summit-301915405.html[3] Medivir reports promising interim data, including a first complete response in phase 1b/2a HCC study with fostrox in combination with Lenvima. Retrieved September 4，2023，from https://www.prnewswire.com/news-releases/medivir-reports-promising-interim-data-including-a-first-complete-response-in-phase-1b2a-hcc-study-with-fostrox-in-combination-with-lenvima-301916719.html[4] Beam Therapeutics Announces First Patient Dosed in Phase 1/2 Trial of BEAM-201 in Relapsed, Refractory T-ALL/T-LL. Retrieved September 5，2023，from https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-first-patient-dosed-phase-12-trial[5] Wave Life Sciences Announces Submission of First Clinical Trial Application for WVE-006, the First-ever RNA Editing Clinical Candidate, and Plans for Upcoming Virtual “R&D Day”. Retrieved September 5， 2023，from https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-submission-first-clinical-trial[6] Nykode Therapeutics Announces FDA Approval of IND for VB-C-04, a Trial of VB10.16 in HPV16-Positive Cervical Cancer. Retrieved September 5，2023，from https://www.globenewswire.com/news-release/2023/09/05/2736940/0/en/Nykode-Therapeutics-Announces-FDA-Approval-of-IND-for-VB-C-04-a-Trial-of-VB10-16-in-HPV16-Positive-Cervical-Cancer.html[7] Oncorena's first patient treated with ONC175 (orellanine) in clinical phase I/II trial in patients with metastatic renal cancer and dialysis at Karolinska University Hospital in Stockholm, Sweden r. Retrieved September 5，2023，from https://www.prnewswire.com/news-releases/oncorenas-first-patient-treated-with-onc175-orellanine-in-clinical-phase-iii-trial-in-patients-with-metastatic-renal-cancer-and-dialysis-at-karolinska-university-hospital-in-stockholm-sweden-301916755.html[8] invIOs starts further clinical trial of APN401, novel cell therapy against solid cancers, and secures major Austrian grant funding. Retrieved September 5，2023，from https://www.globenewswire.com/en/news-release/2023/09/05/2736966/0/en/invIOs-starts-further-clinical-trial-of-APN401-novel-cell-therapy-against-solid-cancers-and-secures-major-Austrian-grant-funding.html[9] Plus Therapeutics Initiates Part B of ReSPECT-LM Phase 1/2a Trial for Leptomeningeal Metastase. Retrieved September 5，2023，from https://ir.plustherapeutics.com/news-releases/news-release-details/plus-therapeutics-initiates-part-b-respect-lm-phase-12a-trial[10] uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-260 Gene Therapy for Refractory Mesial Temporal Lobe Epilepsy. Retrieved September 5, 2023，from https://www.globenewswire.com/news-release/2023/09/05/2737179/0/en/uniQure-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-AMT-260-Gene-Therapy-for-Refractory-Mesial-Temporal-Lobe-Epilepsy.html[11] IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA. Retrieved September 5，2023，from https://www.businesswire.com/news/home/20230906544525/en[12] Hoth Therapeutics Receives Approval to Commence Cohort 2 in Clinical Trial of BioLexa for the Treatment of Atopic Dermatitis Patients. Retrieved September 6，2023，from https://www.prnewswire.com/news-releases/hoth-therapeutics-receives-approval-to-commence-cohort-2-in-clinical-trial-of-biolexa-for-the-treatment-of-atopic-dermatitis-patients-301398082.html[13] Tango Therapeutics Announces FDA Clearance of Investigational New Drug Application for TNG348, a Novel USP1 Inhibitor for the Treatment of BRCA1/2-mutant and Other HRD+ Cancers. Retrieved September 6，2023，from https://www.globenewswire.com/news-release/2023/09/06/2738183/0/en/Tango-Therapeutics-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-TNG348-a-Novel-USP1-Inhibitor-for-the-Treatment-of-BRCA1-2-mutant-and-Other-HRD-Cancers.html[14] PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1. Retrieved September 7，2023，from https://www.globenewswire.com/en/news-release/2023/09/06/2738854/0/en/PepGen-Inc-Announces-Clearance-of-CTA-by-Health-Canada-to-Begin-the-FREEDOM-DM1-Phase-1-Study-of-PGN-EDODM1-in-Patients-with-Myotonic-Dystrophy-Type-1.html[15] GlycoMimetics Announces First Cohort Dosed in Human Phase 1a Study of GMI-1687. Retrieved September 7，2023，from https://www.businesswire.com/news/home/20230906777561/en/[16] SpliSense Successfully Completed Phase 1 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis. Retrieved September 7，2023，from https://www.prnewswire.com/news-releases/splisense-successfully-completed-phase-1-study-of-spl84-rna-based-therapy-for-the-treatment-of-cystic-fibrosis-301918897.html[17] Jnana Therapeutics Presents Additional Data from Phase 1a Clinical Study of JNT-517 at SSIEM Annual Symposium. Retrieved September 7，2023，from https://www.globenewswire.com/news-release/2023/08/31/2735099/0/en/Jnana-Therapeutics-Presents-Additional-Data-from-Phase-1a-Clinical-Study-of-JNT-517-at-SSIEM-Annual-Symposium.html[18] Phanes Therapeutics Announces First Patient Dosed in Phase 1 Study of PT217 for Small Cell Lung Cancer and Other Neuroendocrine Cancers Expressing DLL3. Retrieved September 8，2023，from https://www.prnewswire.com/news-releases/phanes-therapeutics-announces-first-patient-dosed-in-phase-1-study-of-pt217-for-small-cell-lung-cancer-and-other-neuroendocrine-cancers-expressing-dll3-301920004.html[19] Actinium Pharma Presents Survival Data from Extended Follow-Up from Actimab-A + CLAG-M Trial and Preclinical Data Supporting Program Expansion with FLT3 Inhibitor Combinations at SOHO. Retrieved September 8，2023，from https://www.prnewswire.com/news-releases/actinium-pharma-presents-survival-data-from-extended-follow-up-from-actimab-a--clag-m-trial-and-preclinical-data-supporting-program-expansion-with-flt3-inhibitor-combinations-at-soho-301920692.html[20] DS-3939 Enters Clinical Development in Patients Across Several Types of Advanced Solid Cancers. Retrieved September 8，2023，from https://www.businesswire.com/news/home/20230906178594/en/[21] Carrick Therapeutics Announces U.S. FDA Clearance of IND for CT7439, a First-In-Class Inhibitor of CDK12/13. Retrieved September 8，2023，from https://www.globenewswire.com/news-release/2023/09/07/2739242/0/en/Carrick-Therapeutics-Announces-U-S-FDA-Clearance-of-IND-for-CT7439-a-First-In-Class-Inhibitor-of-CDK12-13.html[22] Opus Genetics Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5. Retrieved September 8，2023，from https://www.globenewswire.com/news-release/2023/09/07/2739303/0/en/Opus-Genetics-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-of-Gene-Therapy-OPGx-LCA5-in-Patients-with-Rare-Inherited-Retinal-Disease-LCA5.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

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