STAT November 14, 2025
A new FDA proposal would bring breakthrough treatment to countless families that need it
In May, news broke of a biomedical first: the on-demand design and clinical use of a personalized gene editor for a baby boy born with a rare, severe genetic condition. At the STAT Summit in October, the child’s treating physician from the Children’s Hospital of Philadelphia (CHOP) and the lead on developing the gene editor for him shared the stage with him and his parents. The video of a happily squirming 1-year-old brought tears of joy to the eyes of many.
A sobering reminder of the long road ahead toward more such videos was shared in these pages by Celena Lozano, whose son lives with a...
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