Shanghai-based Rona Therapeutics Inc. announced receiving approval from the US Food and Drug Administration (FDA) to initiate a Phase II clinical study for its RN0361, an APOC3-targeted siRNA therapeutic. The study will assess the drug’s efficacy and safety in patients with severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS).
Drug Mechanism and Platform
RN0361 leverages Rona’s proprietary GAIA siRNA conjugate and oligo chemistry platform. This platform enables subcutaneous administration, ensuring robust and durable hepatocyte-specific silencing. The drug’s optimized chemical modifications allow for sustained gene silencing, significantly reducing APOC3 mRNA and protein expression.
Clinical Significance
The initiation of this Phase II study marks a crucial step forward in addressing these serious lipid disorders. Current treatment options for SHTG, mixed dyslipidemia, and FCS are limited, and there remains a significant unmet need for effective therapies.-Fineline Info & Tech
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