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drugset / Trial / NCT06024876

A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

NCT06024876 ↗

Phase 0 Completed 5 enrolled CorrectSequence Therapeutics Co., Ltd First Affiliated Hospital of Guangxi Medical University · collab

NaSingle-groupOpen-labelTreatment

Summary

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.

Timeline

Start: 2023-08-26
Primary completion: 2025-07-01
Completion: 2025-07-01

Publications

Lai Y, Liu R, Wang L, Ma XK, Li Y, Yang G, Shi L, Guo YL, Wei Z, Zhou X, Xu W, Hou Y, Miccio A, Yang B, Mou X, Yang L, Chen J. Clinical application of base editing for treating beta-thalassaemia. Nature. 2026 May;653(8115):923-932. doi: 10.1038/s41586-026-10342-9. Epub 2026 Apr 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CS-101	Cell therapy	—	—

Indications