Beta-thalassemia, also called Cooley’s Anemia, is a blood disease that effects thousands of Americans. In a major step toward a curative therapy, rare disease drug development firm Errant Gene ...

(CNN) — The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease. The groundbreaking ...

After approving Casgevy (exagamglogene autotemcel) in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat ...

While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell transplantation (HSCT) remains the only widely accessible curative option for this ...

Agios’ Pyrukynd is FDA-approved for pyruvate kinase deficiency but generated modest sales of $8.6 million in Q2 2024. Mitapivat shows promise in treating SCD and thalassemia, with ongoing Phase 3 ...