The executive director of the nonprofit patient advocacy group the Sickle Cell Consortium was diagnosed with sickle cell disease at age 3. Because of it, she’s had heart problems, had her hips replaced, and experienced serious pain all her life.
Bailey told the US Food and Drug Administration’s independent advisory committee Tuesday that she believes a cutting-edge therapy that is currently under review offers the sickle cell community something many haven’t ever had before: hope.
“Hope is on the horizon, and we are looking toward this hope for a change of the lives that we are living of excruciating pain,” Bailey told the FDA committee on Tuesday.
The independent committee is helping the FDA think through how it should evaluate a treatment called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease with no universally successful treatment.
This was an ongoing discussion with no vote or decision about the therapy, but the discussion likely moves the US one step closer to approving a groundbreaking new treatment that uses gene editing.