JWK001: First AAV Gene Therapy Using a Two-Plasmid Packaging System Receives IND Approval for nAMD

On February 22, 2024, the Investigational New Drug (IND) application for "JWK001 Injection," a Class I innovative gene therapy independently developed by Chengdu Jinweike Biotechnology Co., Ltd. ("Jinweike") for neovascular age-related macular degeneration (nAMD), was successfully approved by the National Medical Products Administration (NMPA).

Image Source: CDE Official Website

nAMD is an irreversible ophthalmic disease that severely affects vision and can even lead to blindness. Its main pathological features include choroidal neovascularization (CNV) or retinal neovascularization in the macular area, accompanied by possible changes such as retinal edema, retinal exudation, hemorrhage, and scarring. Vascular endothelial growth factor (VEGF) has been proven to play a significant role in the formation of new blood vessels in nAMD. Currently, wet age-related macular degeneration has become the third leading cause of blindness globally after glaucoma and cataracts, with the number of patients worldwide continuously rising.

JWK001 Injection is the first AAV gene therapy new drug to adopt a "two-plasmid packaging system." The two-plasmid suspension HEK293 cell packaging technology independently developed by Jinweike Biotechnology significantly improves AAV packaging efficiency and reduces production costs. Jinweike's CMC process is stable and cost-effective, offering significant advantages during both clinical and commercial stages, greatly enhancing patient accessibility.

Based on the self-developed "two-plasmid packaging system" technology platform, multiple AAV gene therapy new drugs developed by Jinweike Biotechnology are undergoing IIT research at West China Hospital of Sichuan University. The indications include ophthalmic, genetic metabolic, and neuromuscular diseases.

JWK001 Injection achieves sustained and efficient expression of anti-VEGF protein in retinal cells through an AAV vector carrying a newly self-designed anti-VEGF protein expression cassette. This gene therapy approach avoids risks associated with traditional nAMD treatments, such as damage to ocular tissues caused by repeated intravitreal injections and poor patient compliance, aiming for a single treatment with lifelong effectiveness.

Editor: Muyan

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