Leveraging the proprietary Youngen's KARDIA SHUTTLE™ delivery platform and next-generation site-specific modification and conjugation technology for AOC, Youngen overcomes the delivery limitations of traditional RNAi therapeutics, enabling efficient, precise and safe siRNA delivery to the myocardium and other extrahepatic target tissues. Our cardiac-focused portfolio specifically targets root-cause pathogenic genetic aberrations and pathological signaling pathways responsible for hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM) and heart failure with preserved and reduced ejection fraction (HFpEF and HFrEF).
We currently have five programs within our cardiovascular portfolio. Groundbreaking progress has been made in our candidate pipeline for PLN mutation-induced cardiomyopathy. By silencing key pathogenic genes in cardiac tissues, our candidates effectively reverse myocardial structural abnormalities, suppress adverse ventricular remodeling, and restore cardiac systolic and diastolic function, striving to develop innovative first-in-class RNAi therapeutics for intractable cardiovascular disorders. Several core cardiovascular candidates have entered the preIND stage, and multiple lead programs have launched investigator-initiated trials (IITs), accelerating the clinical translation and clinical validation of cardiac RNAi therapies.
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