Breaking Through the Myocardial Delivery Bottleneck: Small Nucleic Acid Technology Opens a New Era of Precision Treatment for Cardiomyopathy

On September 19–20, the 2026 China Cell and Gene Therapy (CSGCT) Conference was held at the Exhibition and Trading Center of the Zhongguancun National Independent Innovation Demonstration Zone in Beijing. Dr. Gao Qi, Vice President of Hotgen Biotech and co-founder of Youngen Biotech, attended the conference, delivered a keynote report titled "Small Nucleic Acid Technology Opens a New Era of Precision Treatment for Cardiomyopathy" at the Forum on Delivery Breakthroughs and Industrialization Ecosystem for Small Nucleic Acid Drugs, and engaged in in-depth dialogue with multiple industry experts on the topic of "Technical Bottlenecks and Clinical Breakthroughs in Extrahepatic Delivery of Small Nucleic Acid Drugs."

Inherited cardiomyopathy is caused by genetic mutations and includes subtypes such as hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy, arrhythmogenic cardiomyopathy, and transthyretin amyloid cardiomyopathy (ATTR-CM). It is an important cause of sudden death and heart failure in young and middle-aged adults. There are approximately 4.7 to 5 million active cases globally, with 300,000 to 370,000 deaths annually; in China, there are over 7 million potential patients, of whom 85%–90% remain undiagnosed. Existing treatment approaches remain at the level of "symptomatic intervention"—beta-blockers, ACEI/ARB anti-heart-failure drugs, and implantable defibrillators can only improve symptoms and cannot block disease progression at its root.

Cardiomyopathy treatment has long been constrained by two major technological barriers: target druggability challenges and myocardial delivery bottlenecks. None of the currently approved siRNA drugs target cardiomyopathy; the core bottleneck is not the target mechanism, but the lack of myocardial-targeted delivery technology. During on-site exchanges, Dr. Gao Qi described the application prospects of Kardia Shuttle^®. In her view, the significance of a delivery platform goes beyond "getting the drug into the myocardium": Kardia Shuttle^® is responsible for safely and efficiently delivering the drug to myocardial cells, while precision cardiac medicine directly targets the underlying genetic mutations driving disease progression. Only by combining the two can the etiology be intervened at the molecular source. Currently, more than half of familial cardiomyopathy cases can identify clinically actionable mutations through genetic testing. Once the delivery platform is established, genotype identification can be directly translated into a starting point for treatment. With the addition of AI-assisted sequence design, large-scale population genomics, and wearable ECG monitoring, Kardia Shuttle is expected to expand from a myocardial delivery platform into a precision cardiology foundation covering multiple targets and multiple cardiomyopathy subtypes, shifting the intervention point from "control after symptom onset" to "interception before symptom onset," and advancing inherited cardiomyopathy toward becoming preventable and treatable.

With its original technological breakthroughs in the field of small nucleic acid myocardial-targeted delivery, Youngen Biotech was honored with the "Innovation Visionary Enterprise" award at the conference. This award is both a high affirmation of Youngen Biotech's commitment to original innovation and deep cultivation of the small nucleic acid myocardial-targeted delivery technology pathway, and industry recognition of the platform value of Kardia Shuttle^®.