Stopped: We have discontinued our 1st generation HD program with the VYTAL Study \& have initiated a 2nd generation program using a novel, proprietary AAV capsid that may enable intravenous administration \& achieve widespread distribution to affected tissue.
This is the first clinical study of VY-HTT01, a gene therapy for early-stage Huntington's Disease (HD) patients. The primary goal of this trial is to evaluate the safety and tolerability of VY-HTT01. This study is a first in human study, Phase 1b, open-label, randomized, multicenter, dose escalation study with a delayed treatment control arm.
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Incidence and type of AEs
Timeframe: Collected for duration of study, average of 1 year after treatment