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A single dose of Rona Therapeutics' RN0361 reduced triglycerides by up to 69% in people with high levels, with no serious side effects.
Rona Therapeutics reported Phase 1 trial results for RN0361, an siRNA therapy targeting Apolipoprotein C3, at the 2025 American Heart Association Scientific Sessions.
A single dose in people with high triglycerides led to sustained reductions—up to 93% in ApoC3 and 69% in triglycerides—for at least six months, along with significant drops in harmful lipoproteins.
The treatment was well-tolerated, with only mild, temporary side effects.
No serious adverse events occurred.
The data support advancing RN0361 into Phase 2 trials for severe hypertriglyceridemia, a condition linked to pancreatitis and heart disease.
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Una dosis única de RN0361 de Rona Therapeutics redujo los triglicéridos hasta en un 69% en personas con niveles elevados, sin efectos secundarios graves.