Clinical data comparison for FDA-approved gene therapies in sickle cell disease
Alexis Leonard, Julie Kanter · 2025
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Description
Alexis Leonard and Julie Kanter published a 2025 comparison of the two gene therapies approved by the US Food and Drug Administration for sickle cell disease. This inherited blood disorder deforms red blood cells and causes severe pain crises. Curative options were historically limited to bone marrow transplant from a matched donor.
In December 2023 the FDA approved lovo-cel and exa-cel, both made from the patient's own edited blood stem cells. The authors compared the trials behind them: design, eligibility, manufacturing requirements, and reported efficacy and safety. Patients treated with lovo-cel had more severe disease at baseline, with a higher median rate of vaso-occlusive events, despite a stricter definition of those events. The comparison helps clinicians counsel patients choosing between the therapies.
Catalog record
| CREATOR | Alexis Leonard, Julie Kanter |
|---|---|
| YEAR | 2025 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 35 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |