logo uniQure

Clinical Update on First Patients in Phase I/II Clinical Trial Gene Therapy for the Treatment of Huntington’s Disease

16 December, 2021: The data support the tolerability of AMT-130, a potential one-time gene-therapy approach for Huntington’s disease


Yesterday, December 16, 2021, uniQure sent out a press release whose 4 highlights are as follows:

1 - The treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up;

2 - Neurofilament Light Chain (NfL) rose, as expected, immediately following surgery and returned to baseline in treated patients;

3 - A total of 19 patient procedures have been performed in the U.S. Phase I/II clinical trial, with higher-dose cohort enrollment expected to be completed by mid-2022;

4 - Screening initiated in European open-label Phase I/II study.

In addition, it was announced that uniQure also plans to initiate a third cohort in the ongoing U.S. Phase I/II clinical trial in the second half of 2022 that will explore the use of alternative stereotactic navigation systems to simplify placement of catheters for infusions of AMT-130. This will be explored in two steps and include up to 18 additional randomized patients who will receive the higher dose.  


To learn ore about this innovative experimental therapy, watch the OFFICIAL VIDEO

Read HERE a short description of the study.

Previous updates from the company: 

HERE - April 6, 2021

HERE  - October 13, 2020

HEREJuly 4, 2019