Gene therapy using modified stem cells for severe blood disorder

Plain-English translation of NCT05762510 on ClinicalTrials.gov ↗ · Source last updated May 2023 · Translation generated August 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This trial is testing a new treatment called LentiRed that uses gene therapy to help people with transfusion-dependent beta-thalassemia—a serious blood disorder where the body cannot make enough healthy red blood cells. Doctors will take some of your own stem cells, modify them in the laboratory using LentiRed to help them produce healthy blood cells, and then transplant them back into your body. The goal is to reduce or eliminate your need for regular blood transfusions.

People with severe thalassemia depend on regular blood transfusions to survive, which can cause serious complications like iron overload and organ damage. This trial exists to see whether this medication can help patients produce their own healthy blood cells and become independent from transfusions.

Do you qualify?

You likely qualify if…

you are between 5 and 35 years old
you have been diagnosed with transfusion-dependent beta-thalassemia and need regular blood transfusions to survive
you are in generally good health (a performance score of 70 or higher)
you have received medical care at a specialized thalassemia center for at least the past 2 years with detailed medical records available
you and your parent or guardian (if under 18) fully understand the study and are willing to sign an informed consent form

You likely don't qualify if…

you have a history of hepatitis B, hepatitis C, HIV, or tuberculosis
you have had a previous stem cell transplant
you have advanced liver disease or kidney disease
you have a history of cancer or certain genetic cancer syndromes in your family
you have severe heart, lung, or other organ damage from iron overload

What participation looks like

If you join this trial, doctors will collect some of your bone marrow or blood stem cells, send them to a laboratory to be modified with the gene therapy, and then give you chemotherapy to prepare your body to receive the modified cells back. You will then receive a transplant of your own modified cells and be monitored closely in the hospital during recovery. Your participation will last 5 years, with regular follow-up visits to check how well the treatment is working and to watch for any side effects.

AI-generated summary from trial data · Aug 19, 2026 · Not medical advice

Trial locations(1 site)

China

The affiliated hospital of guangxi medical university, Nanning, Guangxi