10月6日,恰逢欧洲基因与细胞治疗学会年会(ESGCT)及西班牙基因与细胞治疗学会双年会前夕,科金生物两位科学创始人——Jose-Carlos Segovia教授(西班牙马德里 CIEMAT-CIBERER 研究所 / FIIS-FJD教授)以及Matthew Porteus教授(美国斯坦福大学教授,现任美国ASGCT主席)联合组织了一场以 “同源重组(HDR):未来发展之路” 为主题的一日专题研讨会,并吸引了多国学者协助支持,David Rawlings, University of Washington, Seattle, USA; Paula Cannon, University of Southern California,g Los Angeles, USA; Ayal Hendel, Bar-Ilan University, Ramat-Gan, Israel; Justin Eyquem, University of California San Francisco, San Francisco, USA; Alessia Cavazza, University College London, London, UK; and Rasmus Bak, Aarhus University, Aarhus, Denmark等著名学者纷纷参会。此次研讨会在西班牙塞维利亚的ESGCT会场召开,得到了BE pharma, Synthego,阿斯利康,touchlight等公司的赞助支持。
On October 6th, just before the Annual Meeting of the European Society of Gene and Cell Therapy (ESGCT) and the Biennial Meeting of the Spanish Society of Gene and Cell Therapy, two scientific founders of DanausGT—Professor Jose-Carlos Segovia (from CIEMAT-CIBERER Institute / FIIS-FJD, Madrid, Spain) and Professor Matthew Porteus (from Stanford University, USA, and current President of the American Society of Gene and Cell Therapy, ASGCT)—jointly organized a one-day thematic symposium titled "Homologous Recombination (HDR): The Path Forward". The symposium garnered support from scholars worldwide, with renowned experts in attendance, including David Rawlings (University of Washington, Seattle, USA), Paula Cannon (University of Southern California, Los Angeles, USA), Ayal Hendel (Bar-Ilan University, Ramat-Gan, Israel), Justin Eyquem (University of California San Francisco, San Francisco, USA), Alessia Cavazza (University College London, London, UK), and Rasmus Bak (Aarhus University, Aarhus, Denmark). Held at the ESGCT venue in Seville, Spain, the symposium also received sponsorship and support from companies such as BE pharma, Synthego, AstraZeneca, and Touchlight.
会议详情,点击链接即可查看:https://www.luminaeu.com/hdr-2025
同源重组(HDR)技术目前是编辑人类基因组运用最广泛的方式之一,本次研讨会聚焦 HDR 技术优化与临床应用,为深入探讨如何在所有核酸酶平台上推进并优化 HDR 技术奠定基础,更为 HDR 领域的学术交流与技术合作搭建了重要平台,进一步推动了该技术在临床转化中的发展,为基因编辑治疗相关疾病提供了更多可能。最新临床进展,更证明了科金生物已经在HDR基因精准插入技术的临床应用上走到了世界前沿。
Matthew Porteus教授会上分享
Professor Matthew Porteus shares research progress
会上,Segovia教授代表科金生物公布了自主研发的完整基因替换技术平台针对丙酮酸激酶缺乏症的临床试验DNGT001,全球首例患者的最新临床数据。经过长达一年的随访,获得了良好的安全数据。截至目前,全球有三例患者接受了HDR基因治疗(在不同适应症),科金生物作为全球首个丙酮酸激酶缺乏症临床实验者,用数据奠定了领域先驱者的地位。
On the symposium, on behalf of DanausGT Biotech, Professor Segovia announced the latest clinical data of DNGT001 , a clinical trial for Pyruvate Kinase Deficiency (PKD) based on the company's independently developed full gene replacement technology platform, involving world's first patient. After a one-year follow-up, favorable safety data were obtained. Up to now, three patients worldwide have received HDR gene therapy for different indications. As the global pioneer in conducting clinical trials for PKD, DanausGT Biotech has established its position as a forerunner in the field with solid data.
Jose-Carlos Segovia教授分享最新进展和临床数据
Professor Segovia shares research progress and latest clinical data
科金生物基因替换技术平台可在敲除致病基因的基础上,利用同源重组原理,同时实现在原位精准的Knock in插入正常基因来恢复和增强基因功能。这种一石二鸟的技术在很多疾病领域有独特的优势,突破常规疗法单一性的限制,可同时做到消除病灶和恢复增强,使诸多常规疗法无法治好或者治疗效果不好的疾病有了更好的选择,应用范畴遍及遗传病、癌症、认知障碍症,乃至抗衰老,是基因治疗的理想目标,代表了基因治疗的发展方向。
DanausGT's gene replacement technology platform can, on the basis of knocking out pathogenic genes, utilize the principle of homologous recombination to simultaneously achieve precise in-situ Knock-in of normal genes, thereby restoring and enhancing gene function. This two-in-one technology has unique advantages in many disease areas: it breaks the limitation of the singularity of conventional therapies, can simultaneously eliminate lesions and restore/enhance gene function, and provides a better option for many diseases that are incurable or poorly treated by conventional therapies. Its application scope covers genetic diseases, cancer, cognitive disorders, and even anti-aging. It represents the ideal goal and development direction of gene therapy.
丙酮酸激酶缺乏症(PKD):
丙酮酸激酶缺乏症是一种罕见的常染色体隐性遗传疾病。可导致患者出现慢性溶血性贫血,即红细胞加速破坏。PKLR基因的突变会导致红细胞能量的缺失,其主要表现为PK酶活性降低、ATP生成减少及上游代谢产物的积聚,造成红细胞能量缺失,并加速红细胞的死亡产生溶血性贫血,甚至导致死亡。
Pyruvate Kinase Deficiency (PKD) is a rare autosomal recessive genetic disorder. It can lead to chronic hemolytic anemia in patients, which accelerates the destruction of red blood cells. Mutations in the PKLR gene can lead to a loss of red blood cell energy, mainly manifested as decreased PK enzyme activity, reduced ATP production, and accumulation of upstream metabolites, resulting in red blood cell energy loss and accelerating red blood cell death, leading to hemolytic anemia and even death.
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关 于 科 金 生 物
无锡科金生物科技有限公司(简称:科金生物)成立于 2022 年 1 月,母公司位于江苏无锡,并在西班牙、香港和上海成立研发中心。团队成员都来自诺华,罗氏,药明康德等世界知名药企,并有诺奖团队成员以及两名国家人才带队。
公司专注于细胞与基因治疗,自主研发的完整基因替换技术平台可以精准地插入或者替换基因,从基因层面根治疾病。这种一石二鸟的技术在很多疾病领域有独特的优势,突破常规疗法单一性的限制,可同时做到消除病灶和恢复增强,范畴遍及遗传病、癌症、认知障碍症,乃至抗衰老。
除了疾病领域的应用,基因精准插入,还可以让个体安全的增加新的功能,未来在农业林业,甚至国防领域都会有应用。这是一项充满想象力的技术,期待人们的生活会因为我们的技术变得更加美好。
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