作者: 遗传性血管水肿。HAE NTLA First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema. Intelliaexpects to complete enrollment in 2H'25 and submit a BLA in 2026$Intellia(NTLA)$ 战略聚焦更能成药领域。
NTLA-2001 疗法,通过脂质纳米颗粒 (LNP) 载体,将携带靶向致病基因 TTR 基因的 sgRNA 和优化的 spCas9 蛋白的 mRNA 序列,递送至肝脏。 1期临床数据显示,在6名接受治疗的患者中 (3名患者接受0.1mg/kg剂量,3例患者接受0.3mg/kg剂量) 。接受治疗28天后,两种不同剂量的患者血清中 TTR 蛋白水平分别平均下降52%...
NTLA-2001 疗法,是一种体内基因编辑治疗,通过脂质纳米颗粒(LNP)载体,将携带靶向致病基因TTR基因的sgRNA和优化的spCas9蛋白的mRNA序列,递送至肝脏。 之前发表在 NEJM 的临床数据显示,在这6名接受治疗的患者中,3名患者接受0.1mg/kg剂量,3例...
HAENTLA First Patient Dosed in the HAELO Phase3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema.Intellia expects to complete enrollment in2H25 and submit a BLA in2026$Intellia(NTLA)$战略聚焦更能成药领域。 01-25 ARKK创新投资追踪: ARKK ...
Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the d...
Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion ...