A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
Status:
NOT_YET_RECRUITING
Trial end date:
2029-04-01
Target enrollment:
Participant gender:
Summary
This study adopted an open, single-arm, non-randomized, dose-escalation research design, aiming to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic and immunogenicity characteristics of single and multiple intravenous infusions of DSL101 in patients with Wilson's disease.