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First patient dosed in VAN-2401 phase 1 clinical trial

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Key Takeaways

  • KH658 is a recombinant adeno-associated virus vector encoding a VEGF receptor fusion protein for wet AMD treatment.
  • The VAN-2401 trial is a multi-center, open-label, dose-escalation study assessing KH658's safety, tolerability, and efficacy.
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The VAN-2401 phase 1 clinical trial will evaluate the use of KH658 for the treatment of wet AMD.

Business, Technology, Internet and network concept. virtual display: Clinical trial (Image credit: ©photon_photo/AdobeStock)

(Image credit: ©photon_photo/AdobeStock)

Chengdu Origen Biotechnology Co, Ltd. And Vanotech Ltd. recently announced the dosing of the first patient in the VAN-2401 multi-center phase 1 clinical trial (NCT06825858).1 The VAN-2401 clinical trial is for the evaluation of KH658 for the treatment for patients with wet age-related macular degeneration (wet AMD). KH658 is described as “a recombinant adeno-associated virus vector that encodes a human [vascular endothelial growth factor (VEGF)] receptor fusion protein,” according to a press release.

“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,” said Avner Ingerman, MD, chief medical officer of Vanotech. “KH658 is designed to deliver anti-VEGF continuously to the retina, with the potential to provide sustained levels that can control the disease.”

VAN-2401 is a multi-center, open-label, dose-escalation clinical trial that will assess the safety, tolerability and efficacy of KH658 as a single suprachoroidal space administration of gene therapy for patients with previous treated wet AMD. It will include around 9 patients with wet AMD who have been previously treated and are responsive to anti-VEGF therapy.

“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, MD, a principal investigator in the VAN-2401 trial, and the director of research at Retina Macula Institute of Arizona.

Reference:
1. Morningstar I. Chengdu Origen and Vanotech Announce First Patient Dosed in VAN-2401 Phase 1 Trial of Gene Therapy for Wet Age-Related Macular Degeneration. Morningstar, Inc. Published May 2, 2025. Accessed May 2, 2025. https://www.morningstar.com/news/globe-newswire/9441302/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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