Global Finance News
07 Dec 2025, 18:27
Vertex Unveils First CASGEVY Data in Children 5–11 and Plans Global Regulatory Filings for 2026
Vertex presented the first clinical data showing that CASGEVY, its CRISPR-based gene-editing therapy, produced strong and durable benefits in children ages 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia. All evaluable young patients met key endpoints: sickle cell patients remained free of vaso-occlusive crises for at least 12 months, and thalassemia patients achieved transfusion independence. Safety findings were consistent with expectations from myeloablative conditioning and transplant. Longer-term results in patients 12 and older continued to show sustained remission and transfusion independence. Vertex plans to begin global regulatory submissions for the 5–11 age group in the first half of 2026.
Vertex presented the first clinical data showing that CASGEVY, its CRISPR-based gene-editing therapy, produced strong and durable benefits in children ages 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia. All evaluable young patients met key endpoints: sickle cell patients remained free of vaso-occlusive crises for at least 12 months, and thalassemia patients achieved transfusion independence. Safety findings were consistent with expectations from myeloablative conditioning and transplant. Longer-term results in patients 12 and older continued to show sustained remission and transfusion independence. Vertex plans to begin global regulatory submissions for the 5–11 age group in the first half of 2026.
