Investigational New Drug application for KHN921 received "study may proceed" from the US FDA. KHN921 is a potential "first-in-class" cardiovascular AAV gene therapy being developed for the treatment of hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations
CHENGDU, China and ROCKAWAY, N.J., May 13, 2026 (GLOBE NEWSWIRE) -- Chengdu Origen Biotechnology Co., Ltd. ("Chengdu Origen") and Vanotech Ltd. ("Vanotech") announced today that the US FDA 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-center, 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.
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, M.D., Chief Medical Officer of Vanotech. "With the 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."
About hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations
Hypertrophic cardiomyopathy (HCM) 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 remodeling 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.
About Chengdu Origen and Vanotech
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 (For more information, please visit https://www.clinicaltrials.gov/study/NCT05657301) 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 (For more information please visit https://www.clinicaltrials.gov/study/NCT06825858).
Forward-Looking Statements
This press release contains "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, Chengdu Origen's and Vanotech's research and development plans in connection with its clinical development program and the gene therapy treatments. Such forward-looking statements include inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of Chengdu Origen/Vanotech development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of pandemics and other health emergencies, including their impact on the global supply chain; the uncertainty that our product candidates will receive regulatory approval necessary to initiate human clinical studies; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products. All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. Investors are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements speak only as of the date of this press release. Chengdu Origen and Vanotech undertake no obligation to update any forward-looking statements, whether as a result of new information, future developments or otherwise.
Contact:
matt.schiller@cnkh.com
