CRISPR-SCD-001(CRISPR-SCD-001) - 药物靶点：核酸酶Cas9 x β-globin_在研适应症：终末期肾脏病，肾移植排斥反应_专利_临床

概要

基本信息

药物类型

造血干细胞疗法

别名

靶点

核酸酶Cas9 x β-globin

作用方式

刺激剂、调节剂

作用机制

HBB基因刺激剂(血红蛋白β链基因刺激剂)、核酸酶Cas9调节剂

治疗领域

免疫系统疾病泌尿生殖系统疾病

在研适应症

终末期肾脏病肾移植排斥反应

非在研适应症-

原研机构

University of California, Los Angeles

在研机构

University of California, Los Angeles

非在研机构-

权益机构-

最高研发阶段临床1期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

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关联

2

项与 CRISPR-SCD-001 相关的临床试验

NCT04774536

/ Recruiting临床1/2期IIT

Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR_SCD001 Drug Product).

开始日期2024-09-18

申办/合作机构

The University of California, San Francisco

[+2]

NCT05525507

/ Recruiting临床1期IIT

A Single-armed, Unblinded, Non-randomized Feasibility Study of Hematopoietic Stem Cell Infusion Following a Conditioning Regimen of Total Lymphoid Irradiation (TLI) and Anti-thymocyte Globulin (ATG) in Patients With a Pre-existing, Well-functioning HLA-identical Kidney Transplant

The study seeks to determine if patients with a pre-existing, well-functioning kidney transplant from a HLA-identical living donor can be withdrawn from immunosuppressive medications without compromising allograft function through hematopoietic stem cell (HPSC) infusion from the same donor. HPSC infusion will be preceded by a conditioning regimen of total lymphoid irradiation (TLI) and rabbit anti-thymocyte globulin (rATG).

开始日期2022-12-21

申办/合作机构

University of California, Los Angeles

100 项与 CRISPR-SCD-001 相关的临床结果

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100 项与 CRISPR-SCD-001 相关的转化医学

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100 项与 CRISPR-SCD-001 相关的专利（医药）

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100 项与 CRISPR-SCD-001 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
终末期肾脏病	临床1期	美国	University of California, Los Angeles	2022-12-21
肾移植排斥反应	临床1期	美国	University of California, Los Angeles	2022-12-21