This study is testing a new gene therapy approach for severe sickle cell disease and beta-thalassemia. It involves taking your own blood stem cells (autologous bone marrow derived CD34+ HSPCs) and modifying them in the lab using a technique called BCL11A enhancer targeting Cas9 ribonucleoprotein. These modified cells are then given back to you. The goal is to correct the genetic problem causing your disease. We are looking for people aged 13 to 40 with severe sickle cell disease (HbSS, HbSβ-thalassemia0) or transfusion-dependent beta-thalassemia. The main goal is to see if these modified cells successfully engraft (start working in your body) after 42 days. The study aims to enroll up to 10 participants.

Study design

This is a single-center study, meaning it's conducted at one location, and is open-label, so both you and the study team will know what treatment you are receiving. It is a pilot study focused on safety and feasibility, aiming to enroll a maximum of 14 participants.

What's involved

If you have sickle cell disease, you will receive blood transfusions for 3 months before your stem cells are collected. All participants will have their stem cells collected through a process called apheresis, which may require multiple collections.

Compensation

Not stated in the trial record.

Follow-up

The primary engraftment of the modified cells will be measured at 42 days after the treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.