AMT-162

uniQure's AMT-162, a gene therapy for SOD1-ALS, has received the green light from the IDMC to proceed with enrollment in the second dose cohort of its Phase I/II EPISOD1 trial.

Ultragenyx seeks accelerated FDA approval for UX111, a gene therapy for MPSIII, based on Phase 1/2/3 trial data.

The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant.

uniQure's AMT-130 demonstrated a statistically significant, dose-dependent slowing of Huntington's disease progression in Phase I/II trials, as measured by the cUHDRS.

uniQure has dosed the first patient in its Phase I/II EPISOD1 clinical trial evaluating AMT-162 for SOD1-ALS, a rare form of amyotrophic lateral sclerosis.