The company reported the safety & biomarker data from an ongoing P-I/II trial to evaluate AMT-130 in 10 patients (6 received AMT-130 & 4 with imitation surgical procedure) with HD for 12mos.
The results showed that therapy was found to be well tolerated at the lower dose of 6×1012 vector genomes with no serious AEs @1yr. of follow-up, reduction in mean CSF NfL & 8% increase over baseline in 6 treated patients while remained stable or slightly declined in 4 patients
53.8% mean reduction of mHTT in CSF @12mos. in 4 patients while increased over baseline @ 1/3/6/9mos. & reduced by 16.8% in 3 patients. The additional data includes 24 & 12 mos. follow-ups in the low & high dose US cohort are expected in H1’23
Ref: GlobalNewswire | Image: uniQure
