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Chengdu Origen Biotechnology Co Ltd, announced that the US Food and Drug Administration granted “study may proceed” to the KHN921 IND application. KHN921 is being studied for the treatment of hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations. The proposed multi-centre, open-label, dose escalation and expansion phase I/II study will assess the safety, tolerability and efficacy of a single intracoronary administration of KHN921 gene therapy in adults with symptomatic MYBPC3 mutation-associated HCM.
Investigational New Drug application for KHN921 received “study may proceed” from the US Food and Drug Administration. KHN921 is a potential "first-in-class" cardiovascular AAV gene therapy being developed for the treatment of hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations.
KHN921 is a recombinant replication-deficient adeno-associated virus vector of serotype AAV9 that encodes a gene encoding the MYBPC3 protein. In preclinical studies of HCM disease models, intracoronary infusion of KHN921 resulted in retention of the transgene product in cardiac tissues for prolonged periods and prevented the disease symptoms. These findings may indicate the potential of KHN921 to offer a single administration treatment for HCM patients with MYBPC3 mutation.
KHN921 is an AAV-based gene therapy designed to directly address the root genetic cause of HCM by delivering a functional copy of the MYBPC3 gene and restoring normal myosin-binding protein C expression in cardiomyocytes,” said Avner Ingerman, chief medical officer of Vanotech. “With the US FDA’s IND clearance, we are one step closer to bringing this potentially transformative therapy to patients who currently face limited long-term treatment options for this life-threatening disease.”
Hypertrophic cardiomyopathy is the most common monogenic cardiovascular disorder, affecting approximately 1 in 500 individuals worldwide. It is characterized by unexplained left ventricular hypertrophy, which can lead to heart failure, arrhythmias, and sudden cardiac death, particularly in young adults and athletes. Mutations in the MYBPC3 gene, which encodes cardiac myosin-binding protein C (cMyBP-C) - a key regulator of sarcomere structure and cardiac contraction - are one of the most prevalent genetic causes of HCM. Pathogenic MYBPC3 mutations lead to impaired or truncated protein production, disrupting normal cardiac function and driving the pathological remodelling seen in HCM. Currently, no disease-modifying therapies targeting the underlying genetic cause of MYBPC3-associated HCM have been approved, representing a significant unmet medical need.
Chengdu Origen is a clinical-stage gene therapy company focused on developing gene therapy for unmet medical needs and providing meaningful clinical benefits for patients suffering from genetic and chronic diseases and has comprehensive viral vector manufacturing capabilities and infrastructure.
Vanotech is operating as the sponsor-representative and is responsible for the clinical development program of KH921 in the United States. Vanotech is also currently conducting VAN-2201, a phase 1, multi-center, open-label, dose-escalation study in subjects with neovascular AMD to evaluate safety, tolerability and efficacy of a single administration of KH631 and VAN-2401, a phase I, Open-label, Multicenter, Dose-escalation study to evaluate the safety and tolerability of a single administration of KH658 gene therapy in participants with neovascular AMD.
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