CRISPR Atlas

临床试验数据库

按疾病、技术、阶段、状态和国家筛选基因编辑临床试验。每条记录均链接至其注册登记页面。

14 Atlas 中收录的临床试验。记录来源于公开注册信息、监管文件及申办方公告。某项试验已注册、正在招募或已完成，并不代表该疗法有效。

14 已显示的临床试验

试验	疾病	治疗方法	技术	阶段	状态	申办方	注册库
CLIMB SCD-121 — the trial that led to the first CRISPR medicine approval A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease	Sickle cell disease	Casgevy (exagamglogene autotemcel)	CRISPR-Cas9	Phase II/III	Completed	Vertex Pharmaceuticals	NCT03745287 ↗
CLIMB THAL-111 — the beta thalassemia trial behind the Casgevy approval A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent…	Beta thalassemia	Casgevy (exagamglogene autotemcel)	CRISPR-Cas9	Phase II/III	Completed	Vertex Pharmaceuticals	NCT03655678 ↗
MAGNITUDE — Phase 3 in vivo CRISPR for ATTR cardiomyopathy A Phase 3 Study of Nexiguran Ziclumeran in Participants With Transthyretin Amyloidosis…	ATTR amyloidosis	Nexiguran ziclumeran (nex-z)	CRISPR-Cas9 (in vivo)	Phase III	Active, not recruiting	Intellia Therapeutics	NCT06128629 ↗
MAGNITUDE-2 — Phase 3 in vivo CRISPR for ATTR polyneuropathy A Phase 3 Study of Nexiguran Ziclumeran in Participants With Hereditary Transthyretin…	ATTR amyloidosis	Nexiguran ziclumeran (nex-z)	CRISPR-Cas9 (in vivo)	Phase III	Paused	Intellia Therapeutics	NCT06672237 ↗
HAELO — the first positive Phase 3 result for in vivo gene editing A Phase 3 Study of Lonvoguran Ziclumeran in Adults With Hereditary Angioedema	Hereditary angioedema	Lonvoguran ziclumeran (lonvo-z)	CRISPR-Cas9 (in vivo)	Phase III	Completed	Intellia Therapeutics	NCT06634420 ↗
BEACON — base editing for sickle cell disease A Study of BEAM-101 in Patients With Sickle Cell Disease With Severe Vaso-Occlusive Crises	Sickle cell disease	Risto-cel (BEAM-101)	Base editing	Phase I/II	Active, not recruiting	Beam Therapeutics	NCT05456880 ↗
BEAM-302 — first in vivo correction of a disease-causing point mutation in humans A Study of BEAM-302 in Patients With Alpha-1 Antitrypsin Deficiency	Alpha-1 antitrypsin deficiency	BEAM-302	Base editing (in vivo)	Phase I/II	Recruiting	Beam Therapeutics	NCT06389474 ↗
The first prime-editing therapy in humans A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox…	Chronic granulomatous disease	PM359	Prime editing	Phase I/II	Recruiting	Prime Medicine	NCT06559176 ↗
BRILLIANCE — the first in vivo CRISPR administration to a human organ Single Ascending Dose Study of EDIT-101 in Participants With Leber Congenital Amaurosis…	Inherited blindness	EDIT-101	CRISPR-Cas9 (AAV, subretinal)	Phase I/II	Completed	Editas Medicine	NCT03872479 ↗
The Phase 1/2 study that took lonvo-z into late-stage development A Study of NTLA-2002 in Adults With Hereditary Angioedema	Hereditary angioedema	Lonvoguran ziclumeran (NTLA-2002)	CRISPR-Cas9 (in vivo)	Phase I/II	Completed	Intellia Therapeutics	NCT05120830 ↗
ANTLER — allogeneic CAR-T built with hybrid RNA-DNA guides A Study of CB-010 in Subjects With Relapsed or Refractory B Cell Non-Hodgkin Lymphoma	Lymphoma	CB-010	CRISPR-Cas12a (chRDNA)	Phase I	Recruiting	Caribou Biosciences	NCT04637763 ↗
Allogeneic CAR-T with next-generation potency edits A Study of CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies	Lymphoma	CTX112	CRISPR-Cas9 multiplex	Phase I/II	Recruiting	CRISPR Therapeutics	NCT05643742 ↗
The first US trial of CRISPR-edited T cells in cancer patients NY-ESO-1-Redirected CRISPR-Edited T Cells in Patients With Multiple Myeloma and Sarcoma	Cancer	—	CRISPR-Cas9 multiplex	Phase I	Completed	University of Pennsylvania	NCT03399448 ↗
The first human gene-editing clinical trial Zinc Finger Nuclease Modification of CCR5 in Autologous CD4 T Cells in HIV	HIV	—	Zinc finger nucleases	Phase I	Completed	Sangamo Therapeutics	NCT00842634 ↗

所显示的状态以图谱中的记录为准——请查看各试验页面了解其最近更新日期。注册库状态可能每天变化；ClinicalTrials.gov（美国国家医学图书馆）是在该平台注册试验的权威记录来源。没有注册库编号的试验属于早期研究或非美国注册研究，页面上会有相应标注。

如果您正在寻找可参与的试验 本数据库仅供教育和研究使用。入组资格由试验研究者决定，而非由网站决定，本网站也无法为您转介至任何研究项目。请咨询您的主治医生，并在 clinicaltrials.gov 或您所在国家的官方注册平台上搜索相关信息。

如何读懂一条试验记录

第 I 期

规模较小，通常为首次人体试验。核心问题是安全性与剂量，而非疗效。

第 II 期

规模更大，旨在验证获益信号并收集更多安全性数据。许多有前景的 II 期结果在此阶段并不能得到重现。

第 III 期

注册性试验——规模更大、周期更长，通常是监管审批决策的依据。

长期随访

基因编辑的改变可能是永久性的，因此监管机构通常要求在试验结束后进行数年的随访跟踪。