A gene therapy we funded just entered its first human trial
After a decade of lab work, a treatment targeting one of the most common X-linked genes has reached the clinic for the first time.
A gene therapy that began as a Xara Foundation Discovery Grant has enrolled its first participants in a Phase 1 clinical trial — the furthest any therapy for this condition has ever advanced.
The approach delivers a working copy of the affected gene directly to neurons, aiming to restore the protein that families have gone without for a lifetime. Early animal data showed durable improvements in learning and motor function, and the trial will now test safety and dosing in a small group of adults before expanding to children.
“This is the moment a decade of work has been building toward,” said the lead investigator. “Whatever the result, we will learn something that moves the whole field forward.” Results from the first cohort are expected within eighteen months.