Gene-Editing hope for thalassemia patients: early trial launched

NCT ID NCT06328764

ENROLLING_BY_INVITATION Disease control Sponsor: CorrectSequence Therapeutics Co., Ltd Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial is testing a new gene therapy called CS-101 for people with beta-thalassemia, a blood disorder that often requires lifelong transfusions. The treatment uses the patient's own stem cells, which are modified in a lab to produce fetal hemoglobin, potentially reducing the need for transfusions. Ten participants aged 6 to 35 will be monitored for safety and effectiveness.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: CS-101 (gene-edited stem cells)
What this could lead to: If successful, this could reduce or eliminate the need for regular blood transfusions in people with beta-thalassemia.
What could go wrong: This is a very early, small trial (10 people) focused on safety. The treatment may not work, and there are risks from the stem cell transplant procedure itself.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for BETA-THALASSEMIA are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

beta thalassemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BETA-THALASSEMIA

Contacts and locations

Locations

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.