Chengdu Origen and Vanotech Announce First Patient Dosed in VAN-2401 Phase 1 Trial of Gene Therapy for Wet Age-Related Macular Degeneration

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Clinical trial of KH658 evaluates single suprachoroidal space administration of gene-therapy treatment for wet AMD

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CHENGDU, China and ROCKAWAY, N.J., May 02, 2025 (GLOBE NEWSWIRE) — Chengdu Origen Biotechnology Co., Ltd. (“Chengdu Origen”) and Vanotech Ltd. (“Vanotech”) announced today the first patient dosed in the VAN-2401 multi-center Phase 1 clinical trial evaluating treatment with KH658 for patients with wet Age-related Macular Degeneration (wet AMD). This multi-center, open-label, dose-escalation clinical trial in the U.S. will assess the safety, tolerability and efficacy of KH658 as a single suprachoroidal space administration of gene therapy for patients with previously treated wet AMD.

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KH658 is a recombinant adeno-associated virus vector that encodes a human VEGF receptor fusion protein. In preclinical studies of wet AMD disease models, suprachoroidal space administration of KH658 resulted in retention of the transgene product in the retina for prolonged periods and prevented the disease progression. These findings may indicate the potential of KH658 to offer a single administration treatment for wet AMD patients.

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“Dosing our first patient in the VAN-2401 Phase 1 trial is an important milestone in the advancement of KH658 by exploring the potential of single suprachoroidal space administration of gene therapy as treatment for wet AMD. KH658 is designed to deliver anti-VEGF continuously to the retina, with the potential to provide sustained levels that can control the disease,” said Avner Ingerman, M.D., Chief Medical Officer of Vanotech.

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“I am excited to participate as a principal investigator in this important study. Dosing the first patient is an important milestone in the development of the single-administration gene therapy approach, which may hold promise to our patients in need of frequent treatment for their retinal diseases,” said Mark Barakat, M.D., a principal investigator in the VAN-2401 Phase 1 trial, Director of Research at Retina Macula Institute of Arizona in Scottsdale Arizona.

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About the VAN-2401 Phase 1 Clinical Trial
VAN-2401 is a Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH658 Gene Therapy in Participants with Neovascular Age-related Macular Degeneration. The study is expected to include approximately nine previously treated wet AMD subjects that are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy. For more information, please visit https://www.clinicaltrials.gov/study/NCT06825858

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About Wet AMD
Wet AMD is the rarer form of AMD, characterized by loss of vision due to abnormal blood vessel formation in and under the retina. These blood vessels tend to leak, and the fluid interferes with the normal retinal structure and function, leading to vision loss. Wet AMD is a leading cause of irreversible vision loss or blindness in the United States and Europe, especially if left untreated. It is estimated that ~1.5 million people living with wet AMD in the United States.

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Anti-VEGF therapies have become the standard of care for wet AMD. Anti-VEGF treatments reduce leakage and fluid accumulation, thereby restoring normal retinal architecture and function, in many patients. Anti-VEGF agents often require frequent, repeat injections to maintain efficacy and control the disease.