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US FDA clears gene therapy KHN921 for hypertrophic cardiomyopathy linked to MYBPC3 mutations

Market News
13 May 2026
GlobeNewsWire
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Bullish
US FDA clears gene therapy KHN921 for hypertrophic cardiomyopathy linked to MYBPC3 mutations

Chengdu Origen and Vanotech announced that the US FDA has approved the Investigational New Drug application for KHN921, a gene therapy targeting hypertrophic cardiomyopathy (HCM) caused by MYBPC3 mutations. This Phase I/II trial will test the safety and efficacy of a single dose of KHN921, designed to restore normal heart muscle protein function by delivering a functional MYBPC3 gene. HCM is a serious genetic heart condition with limited treatment options, and KHN921 could offer a transformative therapy addressing the root cause. The companies are advancing clinical development in the US following this clearance.

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