CS-101 injection

GENETIC3 trials

Sponsors

Children's Hospital of Fudan University, CorrectSequence Therapeutics Co., Ltd

Conditions

Beta-ThalassemiaBeta-Thalassemia Majorβ-thalassemia Major

Early Phase 1

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

CompletedNCT06065189

Children's Hospital of Fudan UniversityBeta-Thalassemia

Start: 2023-11-07End: 2025-08-12Updated: 2026-05-15

Phase 1

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

CompletedNCT06291961

CorrectSequence Therapeutics Co., LtdBeta-Thalassemia Major

Start: 2024-04-18End: 2025-11-17Updated: 2026-02-10

Phase 2

A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major

Not yet recruitingNCT07489196

CorrectSequence Therapeutics Co., Ltdβ-thalassemia Major

Start: 2026-04-05End: 2028-07-31Target: 20Updated: 2026-03-24

Related Papers

Clinical base editing for β-hemoglobinopathies across different genetic backgrounds.

2026-09-07

Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: An autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)

Blood2025-11-03

Rapid, Efficient and Durable Fetal Hemoglobin Production Following CS-101 Treatment in Transfusion-Dependent β-Thalassemia Participants: An Autologous, Ex Vivo Edited CD34+ Stem Cell Product Using the Innovative Transformer Base Editor (tBE)

Blood2024-11-051 citations