gene therapy

uniQure announced the dosing of the first two patients in its European open-label Phase Ib/II clinical trial of AMT-130, a potential one-time gene-therapy approach for the treatment of Huntington’s disease. The clinical trial is taking place at several sites in Poland, the United Kingdom and Germany.

On the last February 2, 2022 uniQure sent out a press release with new updates on their gene therapy for Huntignton 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;