A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)

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.

Timeline

Start

2025-12-31

Primary completion

2028-04

Completion

2029-04

Drugs

Indications