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

Summary

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

Timeline

Start

2024-04-18

Primary completion

2025-11-17

Completion

2025-11-17

Outcome

Met primary endpoint

release “Their paper describes how all five patients infused with CS-101, including three with the β0/β0 of β0/β0-like genotype, discontinued transfusions within one month.” correctsequence.com ↗

release “All patients treated in Phase I have maintained transfusion independence for more than one year” correctsequence.com ↗

Drugs

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

Indications