Warm congratulations to GeneVector on the IND acceptance of its first AAV gene therapy pipeline using a two-plasmid packaging system

On November 30, 2023, the Investigational New Drug (IND) application for JWK001 Injection, a Category 1 innovative gene therapy drug independently developed by Chengdu Jinweike Biotechnology Co., Ltd. (“Genevector”) for the treatment of neovascular age-related macular degeneration (nAMD), was accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) under acceptance number CXSL2300827.

nAMD is an irreversible ophthalmic disease that severely impairs vision and may even lead to blindness. It is mainly characterized by choroidal neovascularization (CNV) in the macular region or retinal neovascularization, and may also be accompanied by retinal edema, retinal exudation, hemorrhage, scarring, and other pathological changes. Vascular endothelial growth factor (VEGF) has been proven to play an important role in neovascularization associated with nAMD. At present, wet age-related macular degeneration has become the third leading cause of blindness worldwide after glaucoma and cataracts, with the global patient population continuing to rise.

JWK001 Injection is the first novel AAV gene therapy drug to use a two-plasmid packaging system. Genevector’s independently developed two-plasmid suspension HEK293 cell packaging technology improves packaging efficiency by 10-fold compared with the three-plasmid system, significantly reducing production costs. Genevector’s CMC process is stable and cost-effective, giving it clear advantages in both the clinical and commercialization stages and greatly improving patient accessibility.

Based on its independently developed two-plasmid packaging system technology platform, six AAV gene therapy candidates developed by Genevector have obtained ethics approvals and are currently undergoing investigator-initiated trials (IITs) at West China Hospital of Sichuan University. The indications cover ophthalmology, inherited metabolic diseases, and neuromuscular diseases.

JWK001 Injection is designed with an independently innovative multi-target gene expression cassette, expanding coverage to patients who are insensitive to single-target therapies and improving therapeutic efficacy. JWK001 Injection offers advantages such as a low effective dose and a favorable safety profile, and has been granted a national invention patent.

JWK001 Injection enables sustained and efficient expression of an anti-VEGF protein in retinal cells by delivering an AAV vector carrying a newly and independently designed anti-VEGF protein expression cassette. This gene therapy approach can avoid the risks associated with conventional nAMD treatment, such as ocular tissue damage caused by repeated intravitreal injections and poor patient compliance, thereby achieving the goal of a one-time treatment with long-term efficacy.

Before receiving JWK001, the first patient had an annualized anti-VEGF injection frequency of 10.18 injections. After treatment with JWK001, no supplemental anti-VEGF therapy was required within 20 weeks.

At 20 weeks after administration, the best corrected visual acuity (BCVA) of the first subject increased from 49 letters to 61 letters, representing an improvement of 12 letters as measured by the ETDRS visual acuity chart.

At 20 weeks after administration, the central retinal thickness of the first subject remained stable.

Dr. Yang Yang, co-founder and CEO of Genevector, stated: “Based on the continuous follow-up results of the first subject in the low-dose group of JWK001 Injection, JWK001 Injection has demonstrated good safety and tolerability, as well as a low effective dose. Patients require only a single administration without adjunctive therapy, bringing significant convenience to patients. The IIT clinical results will lay a solid foundation for advancing our Phase I clinical trial. Once the IND is approved, we will accelerate clinical progress so that more patients can benefit.”

PackGene Biotech is honored to support the progress of Genevector’s AAV gene therapy program. For its IND pipeline, PackGene provided one-stop analytical testing services, including method development and validation, release testing, stability testing, and characterization studies. PackGene also provided comprehensive testing and release support for products from small-scale to pilot-scale production, accelerating the clinical trial application for the novel gene therapy drug JWK001 Injection. Looking ahead, PackGene Biotech will continue to uphold its mission of “making gene therapy affordable for the public,” further deepen its development of vector processes and technologies, provide one-stop CMC solutions for drug innovation, and support the regulatory submission of gene therapy products.