FDA Approves CRISPR Gene Therapy for Young Children with Sickle Cell Disease

The U.S. FDA has granted supplemental approval for Casgevy, the first CRISPR-based gene therapy, to treat sickle cell disease and beta-thalassemia in patients as young as two years old, marking a significant expansion of this groundbreaking treatment.

The top 3

1. Global 1st CRISPR Human Trial: In November 2016, a Chinese team performed the world's first CRISPR human trial on a patient with aggressive lung cancer.
2. CRISPR Nobel Laureates: Emmanuelle Charpentier and Jennifer Doudna received the 2020 Nobel Prize in Chemistry for developing the CRISPR/Cas9 gene-editing tool.
3. CRISPR Future Applications: CRISPR holds promise for treating HIV, cardiovascular diseases, Duchenne muscular dystrophy, and enhancing agricultural crops.

Sources

• ubiehealth.com
• fda.gov

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