Posted 11/1/2023, 9:16:00 PM
FDA Panel Reviews Revolutionary Gene Editing Treatment for Sickle Cell Disease, But Hurdles Remain for Widespread Adoption
- FDA panel discusses Crispr's gene editing drug for sickle cell disease, exa-cel
- Patients report major decreases in painful sickle cell episodes after treatment
- Analysts expect FDA approval as most likely outcome on December 8
- Uptake initially limited due to infrastructure capacity constraints
- Patients may opt for chronic treatments over one-time gene editing due to costs
