Gene therapy company uniQure has announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130 for treatment of Huntington’s disease.
According to a company release, six patients enrolled in the study received AMT-130 and four underwent an imitation surgical procedure. At the 12-month follow-up, measurements of cerebrospinal fluid NfL, a key biomarker of neuronal damage, increased as expected following the AMT-130 surgical procedure and approached baseline at 12 months.
In addition, measurements of mutant HTT