This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM).
Age 18 - 80 years old (including the critical values), regardless of gender;
Body weight at the time of screening is between 40 - 90kg (including the critical values);
TTR gene mutation is confirmed by genetic testing;
AST, ALT, and TBIL ≤ the upper limit of the normal value (ULN);
Amyloidosis is not caused by TTR protein, such as light chain amyloidosis;
There is meningeal transthyretin amyloidosis;
Allergic to any lipid nanoparticle (LNP) component or has previously received LNP and experienced treatment-related laboratory abnormalities or adverse events;
In the dose escalation stage of the first stage, the use history of Patisiran, Inotersen, and Vutrisiran is excluded;
Sequential
YOLT-201
DrugInfusion of YOLT-201 at Day 1
Safety and Tolerability
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
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