Casgevy is a one-time therapy used to treat children aged 12 years and older having sickle cell disease (SCD) having frequent vaso-occlusive crises (pain) And patient’s having beta thalassemia in need of regular blood transfusions. It is approved by FDA. Casgevy is specifically made for each patient using patient’s own edited blood stem cells, and increases the production of fetal hemoglobin/Hbf which is a special type of hemoglobin.
Will it be beneficial or result in a new mutations? What are your thoughts on this?
The first gene therapy approved by the FDA in the United States was Luxturna, which treats a rare inherited eye disease by fixing a faulty gene and restores vision for some patients. More recently, the FDA also approved the first CRISPR gene therapy called Casgevy for sickle cell disease, showing big progress in genetic medicine
In trials with over 100 patients followed for more than five years, 92.3% stayed free from painful vaso‑occlusive crises (VOCs) for at least 12 consecutive months, with average VOC‑free durations around 28 months and a maximum extending beyond 54 months. Hospitalizations related to VOCs were similarly reduced in 97.4% of patients.
I think to decide whether this is beneficial or not is to keep track of the patient’s progress with time. Regular check ups and medical attention will be necessary to avoid any adverse side effects.
Casgevy is designed to be beneficial by precisely editing a patient’s own stem cells to boost fetal hemoglobin, which reduces disease symptoms in SCD and β-thalassemia.
While the risk of unintended (off-target) mutations exists, current clinical data and FDA review indicate these risks are low and are outweighed by the significant therapeutic benefit with long-term monitoring in place.
At first it is beneficial. But you never know what changes or effects a new invention brings.
In initial stages main motive shoud be it gives better result and improve life
That’s a very informative post, Priya! Casgevy truly marks a milestone as the first FDA-approved gene therapy for sickle cell disease and beta thalassemia. By editing a patient’s own blood stem cells to boost fetal hemoglobin (HbF), it directly addresses the root cause of these conditions rather than just managing symptoms.
Very thought-provoking question. Gene editing offers hope for durable treatment, but careful follow-up is essential to rule out new mutations or late complications.