Clinical Trial Goal
To find out if BEAM-101 gene therapy is safe and works well to treat SCD
You may be able to join this trial if you:
- Are 18 – 35 years old
- Have SCD
- Had 4 or more vaso-occlusive events in the last 2 years. This may include pain crises, acute chest syndrome, and other complications
- Have not had a blood or marrow transplant (BMT)
- Do not have a matched sibling donor for BMT
- Agree to have other standard tests done to see if you can be in the clinical trial
Trial Details
First, you’ll have apheresis, a process to collect, separate and save your blood-forming cells.
Doctors will treat your blood-forming cells in a lab with gene therapy. The therapy will edit the blood-forming cells to help your body make healthy red blood cells.
You’ll get standard chemotherapy (chemo) to make room for the treated blood-forming cells.
Then, your treated blood-forming cells, BEAM-101, are given back to you through an intravenous (IV) infusion.
You'll have blood tests to see how well the treatment is working. The clinical trial doctors will check your health for up to 2 years.
The Food and Drug Administration (FDA) has not yet approved BEAM-101 gene therapy.
Watch a video about gene therapy
Doctors will treat your blood-forming cells in a lab with gene therapy. The therapy will edit the blood-forming cells to help your body make healthy red blood cells.
You’ll get standard chemotherapy (chemo) to make room for the treated blood-forming cells.
Then, your treated blood-forming cells, BEAM-101, are given back to you through an intravenous (IV) infusion.
You'll have blood tests to see how well the treatment is working. The clinical trial doctors will check your health for up to 2 years.
The Food and Drug Administration (FDA) has not yet approved BEAM-101 gene therapy.
Watch a video about gene therapy
Locations
Sponsors
lead: Beam Therapeutics Inc.
