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Gene Therapy Offers Hope for Sickle Cell Kids
6 Dec, 2025
Summary
- Gene therapy achieved pain-free status for children with sickle cell disease.
- Patients with transfusion-dependent blood disorder became transfusion-free.
- The therapy utilizes Nobel Prize-winning CRISPR gene editing technology.
Vertex Pharmaceuticals has reported significant success with its gene therapy, Casgevy, in younger patients suffering from blood disorders. Data presented indicates that children aged 5 to 11 with sickle cell disease experienced freedom from painful events following treatment. This marks the first clinical data presented for any genetic therapy in this specific pediatric age group for sickle cell disease.
Furthermore, the therapy demonstrated its potential for treating transfusion-dependent beta thalassemia (TDT). Twelve patients with TDT were able to achieve a transfusion-free status for a minimum of 12 consecutive months, with some durations nearing two years. These outcomes bolster the case for broadening Casgevy's approved applications beyond its current use in patients aged 12 and older.
Casgevy employs the groundbreaking CRISPR gene editing technology, often described as molecular scissors, to correct genetic defects. Vertex plans to submit regulatory filings globally in the first half of next year. The company has also received a National Priority Voucher, expediting the health regulator's review process for Casgevy in the 5-11 year age bracket.
