2024年10月8日,AviadoBio和安斯泰来宣布就AVB-101达成一项独家选择权和许可协议。AVB-101是一种基于AAV的基因疗法,用于治疗GRN基因突变额颞叶痴呆(GRN-FTD)患者。 根据协议条款,安斯泰来公司将获得 AVB-101 在 FTD-GRN 和其他潜在适应症的全球独家开发和商业化许可。安斯泰来公司将为 AVB-101 的许可权提供2000...
只有在GRN基因突变的FTD患者的脑脊液中发现GPNMB的表达水平的升高.结论:我们的研究证实了系统生物学方法在研究FTD这种复杂疾病的发生发展机理中的较强的实用性,从小鼠和人类样本两个方面揭示了缺乏PGRN所致的神经系统退行性病变可能由于溶酶体功能异常和神经免疫反应所致,并为寻找GRN基因突变的FTD的生物学标记物或新的...
AviadoBio近日宣布,它已经启动了其研究性基因疗法AVB-101的1/2期ASPIRE-FTD临床研究,旨在评估AVB-101在FTD GRN患者中的安全性和初步疗效,通过丘脑内给药,治疗患有前颗粒蛋白(GRN)基因突变的额颞叶痴呆(FTD)患者。作为一种破坏性的早发性痴呆症,目前没有缓解疾病的治疗方法。AviadoBio公司的首次临床试验启动也表明了...
近日,Denali Therapeutics公司宣布,其合作伙伴武田制药(Takeda)已根据2家公司之间的现有合作协议行使了选择权,共同开发和商业化DNL593(PTV:PGRN)。这是一种脑渗透颗粒体蛋白前体替代疗法,用于治疗携带颗粒体蛋白突变(GRN+)的额颞叶痴呆(FTD-GRN)。 2018年1月,Denali和武田签订了一份合作协议,根据该协议,武田获得了包...
已知颗粒体蛋白(GRN)基因突变或者微管相关蛋白 tau (MAPT) 基因突变与 FTLD 相关。明确 C9orf72 为第三个 FTLD 相关的主要基因之后,Van Langenhove 及其同事开始在每个基因的隐匿突变者队列中进行基因型 - 表型相关性研究,并设计修订分子诊断测试的指南。
In contrast, untreated symptomatic FTD-GRN patients typically experience increasing NfL levels, underscoring the potential of PBFT02 in slowing disease progression. As of December 2024, Passage Bio is progressing to evaluate a 50 percent lower Dose 2 in additional cohorts of FTD-GRN and FTD-C9orf...
The three most common gene mutations that may lead to FTD areGRN,C9orf72, andMAPT. Currently 50,000-60,000 people affected FTD currently affects 50,000 to 60,000 people in the U.S. 40% have an affected relative About 40% of people living with FTD have a family history of a neurode...
Frontotemporal dementia (FTD) is one of the most important neurodegenerative conditions and Granulin (GRN) is one of the major genes associated to the disease. FTD-GRN patients are still orphan for any evidence-based target-therapy approach. Interestingly, it has been recently found that ...
One single-nucleus RNA sequence study targeting the brain cortex of 13 FTD-GRN patients showed severe neurovascular dysfunction and perturbed blood–brain barrier (BBB), posing interest in the vascular function of FTD [1]. White matter hyperintensity (WMH) can reflect neurovascular damage in vivo ...
Amyotrophic lateral sclerosis overlapping with frontotemporal dementia (ALS/FTD) is a fatal and currently untreatable disease characterized by rapid cognitive decline and paralysis. Elucidating initial cellular pathologies is central to therapeutic targe