uniQure paused dosing in the mid- and high-dose cohorts of its Phase 1/2a trial for AMT-191, an AAV gene therapy for Fabry disease, on February 6, 2026, after two patients in the mid-dose group (4×10^13 gc/kg) experienced asymptomatic Grade 3 liver enzyme elevations confirmed as dose-limiting toxicities.1245
Despite the pause, all 11 patients across three dose levels showed elevated alpha-galactosidase A (α-Gal A) enzyme activity, with six discontinuing enzyme replacement therapy (ERT); lower doses showed no serious adverse events.247
GSK's Nucala received EU approval, as noted in recent biopharma updates.6
Gilead announced a label update for its CAR-T therapy, part of the latest biopharma developments.6
Sources:
1. https://www.lqventures.com/lucid-diligence-brief-uniqure-amt-191-fabry-dosing-pause/
2. https://www.fiercebiotech.com/biotech/uniqure-pauses-fabry-gene-therapy-dosing-2-groups-after-toxicities-emerge
4. https://www.clinicaltrialsarena.com/news/uniqure-fabry-disease-gene-therapy-amt-191-phase-i-iia-safety/
5. https://www.biospace.com/drug-development/liver-toxicities-force-pause-on-2-dose-groups-of-uniqures-mid-stage-fabry-study
6. https://firstwordpharma.com/story/7096223
7. https://www.globenewswire.com/news-release/2026/02/06/3233740/0/en/uniQure-Announces-Updated-Preliminary-AMT-191-Phase-I-IIa-Data-Showing-Sustained-Increases-in-%CE%B1-Gal-A-Enzyme-Activity-in-Patients-with-Fabry-Disease.html