Chengdu Origen, Vanotech dose first patient in wet AMD gene therapy trial

Chengdu Origen and Vanotech, both subsidiaries of Chengdu Kanghong Pharmaceutical Group, announced the first wet age-related macular degeneration patient dosed in a phase 1 multi-center clinical trial evaluating treatment with an AAV gene therapy candidate.

The VAN-2401 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. 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.

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. The companies believe KH658 has the potential to offer a single administration treatment for wet AMD patients.

Wet AMD, the rarer form of AMD, is a leading cause of irreversible vision loss or blindness in the U.S. and Europe, especially if left untreated.