其中，在相对成熟的肝靶向递送技术上，炫景生物也建立了GalNAc缀合技术，但不同于同行熟知的“三叉戟”结构，炫景生物设计了新一代GalNAc缀合 ...

点击上方的蓝色“医谷”关注我们近日，悦康药业宣布，其全资子公司杭州天龙药业申报的YKYY015注射液在中国获批一期临床试验，拟开发治疗以LDL-C升高为特征的原发性（家族性和非家族性）高胆固醇血症或混合型高脂血症的患者。根据《中国血脂管理指南（202 ...

10月8日，渤健中国宣布，中国国家药品监督管理局附条件批准托夫生注射液（商品名：凯盛迪）用于治疗携带超氧化物歧化酶1（SOD1）基因突变的肌萎缩侧索硬化（ALS）成人患者。图片来源：NMPA ...

Starting with AATD, in October we delivered a breakthrough in RNA medicine with the first-ever clinical demonstration of RNA ...

RNAi therapeutics have shown great success with multiple US Food and Drug Administration (FDA)-approved products involving N-acetyl galactosamine (GalNAc)-conjugated siRNAs to treat liver-targeted ...

Additionally, Prime Medicine has made significant strides in its gene editing programs, particularly with the unveiling of their GalNAc targeting ligand LNPs, which exhibited high editing efficiency ...

英克司兰是Alnylam基于其递送系统GalNAc研发设计的一款靶向PCSK9的siRNA基因疗法，最初于2020年12月在欧盟获批，用于治疗成人高胆固醇血症及混合性 ...

Wave Life Sciences (WVE)正经历投资者兴趣的显著增长，这从其近期的市场表现中可见一斑。根据InvestingPro数据，该公司股票在过去三个月内实现了195.9%的总价格回报率，年初至今的回报率更是高达214.65%。这与公司报告的临床管线进展和各项目的积极数据相一致。

Prime Medicine’s universal LNP contains a GalNAc-targeting ligand (GalNAc-LNP), a validated mechanism for liver-specific delivery of gene editors. Highlights included: Preclinical data demonstrated ...

Good morning, and welcome to the Wave Life Sciences Third Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen only mode. As a reminder, this call is being ...

Achieved first-ever RNA editing in humans in RestorAATion-2 trial of WVE-006 (GalNAc-AIMer) in alpha-1 antitrypsin deficiency and announced three wholly owned GalNAc-AIMer preclinical programs that ...