Warm congratulations to Jinweike on the acceptance of IND for its first AAV gene therapy pipeline product that employs a two-plasmid packaging system

On November 30, 2023, the IND application for “JWK001 Injection”—a Class I innovative gene therapy for neovascular age-related macular degeneration (nAMD) independently developed by Chengdu Jinweike Biotechnology Co., Ltd. (“Jinweike”)—was accepted by the National Institutes for Food and Drug Control (CDE) under the National Medical Products Administration (NMPA) (Acceptance No.: CXSL2300827).

nAMD is a serious, irreversible ophthalmic disease that can severely impair vision and even lead to blindness. Its main pathological features include choroidal neovascularization (CNV) or retinal neovascularization, and it may also be accompanied by changes such as retinal edema, retinal exudation, hemorrhage, and scar formation. Vascular endothelial growth factor (VEGF) has been proven to play an important role in neovascular formation in nAMD. Currently, wet age-related macular degeneration is the third leading cause of blindness globally after glaucoma and cataracts, and the number of patients worldwide continues to rise.

JWK001 Injection is the first AAV gene therapy new drug to adopt a “two-plasmid packaging system.” Jinweike’s independently developed two-plasmid suspension HEK293 cell packaging technology improves packaging efficiency by 10-fold compared with three-plasmid packaging, substantially reducing manufacturing costs. Jinweike’s CMC process is stable and cost-effective, which will provide clear advantages both in the clinical stage and the commercial stage, greatly improving patient accessibility.

Based on Jinweike’s independently developed “two-plasmid packaging system” technology platform, six AAV gene therapy drug candidates developed by Jinweike have obtained ethics approvals and are currently conducting IIT studies at West China Hospital of Sichuan University. The indications include ophthalmology, genetic/metabolic diseases, and neuromuscular diseases.

JWK001 Injection is designed with an independently innovated multi-target gene expression cassette, increasing coverage for patients who are insensitive to a single target and thereby improving therapeutic efficacy. JWK001 Injection has advantages such as low effective dose and good safety, and it has already obtained authorization for a national invention patent.

JWK001 Injection delivers an AAV vector carrying a newly designed, proprietary anti-VEGF protein expression cassette to enable sustained and efficient expression of the anti-VEGF protein in retinal cells. This gene therapy approach can avoid risks present in traditional nAMD treatments, such as harm to ocular tissues caused by repeated intravitreal injections and poor patient adherence, and it aims to achieve the goal of one treatment with long-lasting effectiveness.

Before receiving JWK001, the annualized number of anti-VEGF injection treatments for the first patient was 10.18 times. After receiving JWK001, within 20 weeks, the patient no longer required any additional anti-VEGF treatment.

At 20 weeks post-dosing, the first subject’s best-corrected visual acuity (BCVA) improved from 49 letters to 61 letters—an improvement of 12 letters (assessed using the EDTRS visual acuity chart).

At 20 weeks post-dosing, the first subject’s central retinal thickness remained stable.

Yang Yang, MD/Researcher, Co-Founder and CEO of Jinweike Biotechnology, stated:
“Based on the ongoing follow-up results of the first subject in the low-dose group, JWK001 Injection has demonstrated good safety and tolerability, with a low onset/effective dose. Patients only need one administration and do not require additional therapy, bringing great convenience to patients. The IIT clinical results will provide a solid foundation for advancing into Phase I clinical development. After IND approval, we will accelerate clinical progress so that more patients can benefit.”

Piping (Pirewin) Biotechnology is honored to support Jinweike’s progress in AAV gene therapy and to provide Jinweike’s IND pipeline with a one-stop analytical and testing service, including method development and verification, release testing, stability testing, and characterization studies. For products from small-scale to pilot-scale production, we provide comprehensive testing and release, thereby accelerating the IND submission for the clinical trial of JWK001 Injection. In the future, Piping Biotechnology will continue to fulfill the mission of “making gene therapy affordable for the people”, and will further focus on the development of vector process and technologies, providing a one-stop CMC solution for drug innovation to support gene therapy drug applications.