BEACON Study is researching a new treatment called BEAM-101 for people with severe Sickle Cell Disease (SCD). SCD is a condition where red blood cells, which carry oxygen, are shaped like a sickle instead of being round. This can cause pain and other health problems. BEAM-101 uses a special kind of cell (CD34+ HSPCs) from the patient’s own body, changes them, and puts them back to help treat the disease. The study is open-label, meaning everyone knows what treatment is being given, and single-arm, meaning everyone gets the same treatment. It is being conducted at multiple centers.
To join, you should:
- Be between ages 12 to 35.
- Have severe SCD with certain types of the disease.
- Have had frequent painful events (at least 4 in the last 2 years) despite treatment.
Some reasons you might not join:
- High levels of fetal hemoglobin (HbF) at the time of screening.
- Previous transplants or available sibling donor.
- History of stroke or certain brain conditions.
The study focuses on safety (no serious side effects) and efficacy (how well it works).
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