Delving into the Latest Updates on 广州辑因医疗科技有限公司 with Synapse

This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

开始日期2023-02-18

申办/合作机构

广州辑因医疗科技有限公司

NCT04925206 / Active, not recruiting临床1期

A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion Dependent β-Thalassaemia

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

开始日期2021-08-17

申办/合作机构

广州辑因医疗科技有限公司

NCT04933825 / Active, not recruitingN/A

An Exploratory,Open-label,and Single Center Study to Evaluate the Safety and Efficacy of ET-02 in Patients With Relapsed or Refractory B-cell Malignancies(NHL/ALL).

The purpose of this study is to evaluate the safety and efficacy of CD19-UCART in patients with r/r B-cell hematological malignancies.

开始日期2021-07-20

申办/合作机构

广州辑因医疗科技有限公司

100 项与广州辑因医疗科技有限公司相关的临床结果

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0 项与广州辑因医疗科技有限公司相关的专利（医药）

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项与广州辑因医疗科技有限公司相关的文献（医药）

Signal transduction and targeted therapy

Highly efficient gene editing and single cell analysis of hematopoietic stem/progenitor cells from X-linked sideroblastic anemia patients

Letter

作者: Zhang, Jingliao ; Wei, Dong ; Zeng, Huimin ; Cheng, Tao ; Zhang, Yingchi ; Shi, Jia ; Yang, Huihui ; Yuan, Pengfei ; Zhu, Xiaofan ; Fang, Riguo

X-linked sideroblastic anemia (XLSA), which is the most common genetic form of congenital sideroblastic anemia, is typically characterized by reduced heme synthesis and the presence of bone marrow (BM) ring sideroblasts containing pathol. iron deposits in the mitochondria.

100 项与广州辑因医疗科技有限公司相关的药物交易

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100 项与广州辑因医疗科技有限公司相关的转化医学

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管线布局

2024年12月23日管线快照

管线布局中药物为当前组织机构及其子机构作为药物机构进行统计，早期临床1期并入临床1期，临床1/2期并入临床2期，临床2/3期并入临床3期

临床1期

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其他

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当前项目

药物(靶点)	适应症	全球最高研发状态

CRISPR/Cas9 基因修饰BCL11A红系增强子的自体CD34+造血干祖细胞 (博雅辑因) ( BCL11A )	输血依赖性地中海贫血更多	临床1期
ET-02 ( CD19 )	急性淋巴细胞白血病更多	临床阶段不明