FDA Approval for Sickle Cell Disease Gene-Therapy | Medical News Bulletin
FDA approved Casgevy and Lyfgenia, gene therapies for Sickle Cell Disease, in December 2023. Casgevy uses CRISPR/Cas 9 to reactivate fetal haemoglobin, while Lyfgenia inserts a new gene for haemoglobin A. Clinical trials showed reduced sickle cell crises, with minor side effects.
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Children ages 12+ with sickle cell disease now eligible for FDA-approved gene therapy Casgevy at Children's Wisconsin, which reduces severe vaso-occlusive crises and transfusion dependence. Casgevy uses CRISPR-modified stem cells to produce fetal hemoglobin, offering fewer side effects than traditional treatments.
Children ages 12+ with sickle cell disease can now receive the FDA-approved gene therapy Casgevy at Children's Wisconsin, which produces fetal hemoglobin to reduce symptoms and transfusion dependence. Casgevy, using CRISPR-modified stem cells, showed 93% of patients free from severe crises for 12+ months and 92% of thalassemia patients transfusion-free. This therapy aims to decrease severe acute events and is particularly beneficial for Black populations, with 1,200 Wisconsin residents affected.
FDA approved Casgevy and Lyfgenia, gene therapies for Sickle Cell Disease, in December 2023. Casgevy uses CRISPR/Cas 9 to reactivate fetal haemoglobin, while Lyfgenia inserts a new gene for haemoglobin A. Clinical trials showed reduced sickle cell crises, with minor side effects.