CRISPR Gene Therapy: “Exa-cel,” a CRISPR-based gene therapy, shows promising results by reducing pain crises and hospitalizations (Cleveland Clinic) (Drugs.com).
New Small Molecule Treatment: A new molecule inducing fetal hemoglobin production offers hope for patients unresponsive to hydroxyurea (ScienceDaily).
CRISPR-Cas9 Trials: Trials editing the HBG1 and HBG2 promoters to boost fetal hemoglobin are showing encouraging outcomes (ScienceDaily).
FDA Reviews: The FDA will decide on two new gene therapies by year’s end, potentially offering curative options (Drugs.com).
