Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle ...

The Italian Medicines Agency's decision has been published in the Official Journal, establishing reimbursement for the revolutionary therapy that frees patients from transfusions. Indeed, the Italian ...

The new Prescription Drug User Fee Act target date is now December 7, 2025. The Food and Drug Administration (FDA) has extended the review period for the supplemental New Drug Application (sNDA) for ...

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta ...

(WASHINGTON — August 27, 2025) – Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or ...

EXTON, PA, Aug. 14, 2025 (GLOBE NEWSWIRE) -- As the first wave of gene therapies for transfusion-dependent β-thalassemia (TDT) continues to roll out in the U.S., a new conversation is emerging within ...

Please provide your email address to receive an email when new articles are posted on . A 69-year-old woman was referred to the retina service for abnormal posterior segment findings. The patient’s ...

Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy.

Editas Medicine reports successful gene editing levels in stem cells, supporting potential treatment for sickle cell disease and beta thalassemia. Editas Medicine announced promising new data on May ...