KH631 / Chengdu Kanghong Pharma - LARVOL DELTA

Bilateral sequential injection of KH631 in rhesus monkeys showed good safety profile (ASGCT 2025) - "The research results indicate that after repeated administration to the contralateral eye, the systemic safety is good and no new adverse reactions emerge compared with the first administration. This provides strong support for repeating the administration to patients in the real world. Disease Focus of Abstract:Ophthalmic Diseases"

Clinical • Age-related Macular Degeneration • Hematological Disorders • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration • CD8 • IL10 • IL2 • IL4 • IL5 • IL6 • PTPRC

KH631 consistently expressed in rhesus monkey eyes over 152 weeks (ASGCT 2025) - "The results demonstrated that KH631 was consistently expressed in rhesus monkey eyes for 152 weeks at all three doses, suggesting that a single subretinal injection of KH631 has the potential to offer a one-time treatment for nAMD patients. Disease Focus of Abstract:Ophthalmic Diseases"

Preclinical • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Evaluating the impacts of pre-existing NAb and ADA in rhesus monkeys on the efficacy of subretinal injection of KH631 (ASGCT 2025) - "As with other gene therapy products administered via subretinal injection, there was no negative effect of pre-existing anti-AAV antibodies on the therapeutic efficacy of KH631. Moreover, the presence of ADA did not impede the efficacy of KH631 as observed in the monkey CNV model. Disease Focus of Abstract:Ophthalmic Diseases"

Clinical • Gene Therapies • Ophthalmology

VAN-2201: Phase 1 study of KH631 gene therapy for the treatment of wet AMD (NCT05657301) (ARVO 2025) - P1 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Gene therapy • P1 data • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

KH631 effectively inhibited the leakage of the CNV model at 87 weeks after subretinal injection (ARVO 2025) - "KH631 employs an adeno-associated virus vector encoding conbercept (an anti-VEGF fusion protein, which was approved in China in 2013) to provide sustained anti-VEGF levels in the retina following a single subretinal injection. Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Gene Therapy for Neovascular AMD (nAMD) by Subretinal Delivery of KH631: a Phase I Study (NCT05672121) (ARVO 2025) - P1/2 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Gene therapy • P1 data • Age-related Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration (clinicaltrials.gov) - P1 | N=25 | Recruiting | Sponsor: Chengdu Origen Biotechnology Co., Ltd.

Gene therapy • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Preclinical evaluation of KH631, a novel rAAV8 gene therapy product for neovascular age-related macular degeneration. (PubMed, Mol Ther) - "Furthermore, sustained transgene expression was observed for more than 96 weeks. These findings suggest that a single subretinal injection of KH631 has the potential to offer a one-time, low-dose treatment for nAMD patients."

Gene therapy • Journal • Preclinical • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD) (clinicaltrials.gov) - P1/2 | N=42 | Recruiting | Sponsor: Chengdu Origen Biotechnology Co., Ltd. | Not yet recruiting ➔ Recruiting | Trial primary completion date: Dec 2023 ➔ Dec 2026

Enrollment open • Gene therapy • Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration (clinicaltrials.gov) - P1 | N=25 | Recruiting | Sponsor: Chengdu Origen Biotechnology Co., Ltd. | Not yet recruiting ➔ Recruiting | Trial completion date: May 2027 ➔ Sep 2027 | Initiation date: Apr 2023 ➔ Aug 2023 | Trial primary completion date: May 2026 ➔ Sep 2026

Enrollment open • Gene therapy • Trial completion date • Trial initiation date • Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Subretinal Delivery of an AAV Gene Therapy Product Significantly Inhibited the Neovascularization in a Laser-Induced CNV Rhesus Model (ASGCT 2023) - "Currently available treatments for neovascular Age-related macular degeneration (wAMD) include Conbercept,Aflibercept, Ranibizumab, Faricimab and Bevacizumab which are antibodies targeting the VEGF axis...The purpose of this study is to develop a therapeutic strategy that have a long-lasting effect with reduced administration frequency, potentially even a single administration.We developed a gene therapy medication (KH631) that encodes anti-VEGF (Conbercept) transgene inside a rAAV8 vector (AAV8-Conbercept)...Laser modeling was conducted 28 days post injection for all the animals in both eyes; Two animals were laser injured and injected immediately with 0.5 mg/eye of Conbercept as positive controls. FL test was performed at week-4 post modeling, images show the representative images of fluorescein fundus angiography (FFA)."

Gene therapy • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD) (clinicaltrials.gov) - P1/2 | N=42 | Not yet recruiting | Sponsor: Chengdu Origen Biotechnology Co., Ltd.

Gene therapy • New P1/2 trial • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Safety and Tolerability Trial of KH631 Gene Therapy for Neovascular AMD (clinicaltrials.gov) - P1 | N=25 | Not yet recruiting | Sponsor: Chengdu Origen Biotechnology Co., Ltd.

Gene therapy • New P1 trial • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration