This Week’s CGT News: Vertex’s Gene Therapies Show Promise in Younger Children
December 10, 2025
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5 min
Two pediatric trials are investigating the gene therapy exagamglogene autotemcel (exa-cel) for children with beta-thalassemia and sickle cell disease, with preliminary results indicating improved safety and efficacy when administered earlier in life. Research has shown that 6 of 13 children treated for beta-thalassemia achieved transfusion independence for 12 months, while 4 of 11 children treated for sickle cell disease remained free from severe vaso-occlusive crises for a year. Meanwhile, advancements in cell therapy are seen with Pan Cancer T's funding for a new breast cancer therapy and FDA approval for Bristol Myers Squibb’s CAR T cell therapy, aiming to address limited treatment options.
1. Exa-cel gene therapy shows promise in pediatric trials for sickle cell and beta-thalassemia. 2. 6 out of 13 treated beta-thalassemia children achieved transfusion independence. 3. 4 out of 11 sickle cell children remained free from severe crises for a year. 4. FDA approved Breyanzi for relapsed marginal zone lymphoma. 5. New funding secured for breast cancer therapy research. 6. Cellipont expanded manufacturing capabilities for cell therapies.
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