China’s first! Congratulations to GeneVector on the clinical trial approval of its gene therapy drug for X-linked retinoschisis

Recently, the investigational new drug (IND) application for JWK002 Injection, a Class 1 innovative gene therapy drug independently developed by Chengdu Jinweike Biotechnology Co., Ltd. (“Genevector”) for the treatment of X-linked retinoschisis (XLRS), was approved by the National Medical Products Administration (NMPA). The clinical trial is expected to be initiated in the near future. As an important strategic partner of Genevector, PackGene Biotech extends its heartfelt congratulations on this significant progress.

JWK002 Injection is the first gene therapy drug for X-linked retinoschisis in China to enter a registrational clinical trial. Previously, JWK002 received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA).

The CMC process for JWK002 Injection adopts Genevector’s independently developed two-plasmid packaging process system. Its suspension HEK293 cell packaging technology is stable and readily scalable, significantly reducing production costs. This process is expected to provide clear advantages in both clinical and commercial stages, greatly improving patient accessibility.

PackGene Biotech is honored to support the advancement of Genevector’s AAV gene therapy drug by providing one-stop analytical testing services for its IND pipeline, including method development and validation, release testing, stability testing, and characterization studies. PackGene provided comprehensive testing and release support for products from small-scale to pilot-scale production, accelerating the acceptance of the clinical trial application for the gene therapy drug JWK002 Injection. Looking ahead, PackGene Biotech will continue to uphold its mission of “making gene therapy affordable and accessible to the public,” deepen its efforts in vector process and technology development, provide one-stop CMC solutions for drug innovation, and support the registration and development of gene therapy drugs.

About X-Linked Retinoschisis

X-linked retinoschisis (XLRS) is an X-linked recessive inherited disease caused by mutations in the retinoschisin 1 (RS1) gene. It is mainly characterized by varying degrees of vision loss, visual field defects, foveal schisis, and reduced b-wave amplitude on electroretinography. XLRS primarily affects males and is usually diagnosed in early childhood. The severity of visual impairment varies among patients. In severe cases, complications such as retinal detachment and vitreous hemorrhage may occur. Current clinical management mainly includes follow-up observation, medications such as carbonic anhydrase inhibitors, and treatment of complications, while no effective clinical treatment is currently available.

About JWK002 Injection

JWK002 Injection is an adeno-associated virus (AAV) gene therapy product independently developed by Genevector. Through systematic design of targeted serotype screening and gene expression elements, JWK002 can efficiently restore RS1 protein expression in retinal cells, thereby improving retinal structure and function in patients.

In a previous investigator-initiated clinical study (IIT) conducted at West China Hospital of Sichuan University, multiple subjects had completed dosing and more than one year of follow-up. The results showed significant improvement in best-corrected visual acuity (BCVA), significant reduction in central retinal thickness (CRT), complete closure of retinoschisis cavities in all treated eyes, and improved retinal photosensitivity in most treated eyes.

Yang Yang, Co-founder and CEO of Genevector, stated:

“We are very pleased that our independently developed JWK002 has entered a registrational clinical trial. This represents another important milestone for the company. Based on the ongoing follow-up results from the IIT clinical study of JWK002, the drug has demonstrated favorable safety and tolerability, with a low effective dose. Patients require only a single administration and no adjunctive therapy, bringing great convenience to patients. Going forward, our team will accelerate clinical development so that more patients can benefit. We would also like to express our special thanks to West China Hospital of Sichuan University, Professor Lu Fang’s ophthalmology team, and Genevector’s shareholders for their strong support.”

About Genevector

Genevector is a biotechnology company dedicated to the development of innovative AAV gene therapy drugs for ophthalmic diseases, neuromuscular disorders, inherited metabolic diseases, and other conditions. Currently, two AAV gene therapy products in its pipeline have entered the registrational clinical stage, while several additional pipelines have entered IIT clinical research.

Under the leadership of Academician Wei Yuquan and Researcher Yang Yang, Genevector has established a 3,000-square-meter R&D and production facility integrating AAV gene therapy drug development, pilot-scale manufacturing, and quality control. The company has built a highly integrated and comprehensive AAV gene therapy “technology chain,” with leading domestic capabilities in gene therapy drug R&D and AAV manufacturing technology. AAV gene therapy products manufactured by Genevector have been evaluated in clinical studies involving more than 100 patients, demonstrating excellent safety and efficacy data.

Genevector’s core management team comes from leading pharmaceutical companies, research institutions, and universities in China and abroad. Its core technical team has expertise in AAV vector design, pilot-scale production, quality research, and clinical medicine, enabling Genevector to operate across the full spectrum from original gene therapy product design to scaled pilot production, medical research, and clinical development.

Leveraging its strong technology platform, Genevector will collaborate with leading industry partners in China and abroad, integrate resources, and improve the accessibility of AAV gene therapy drugs, with the aim of benefiting a broader patient population more quickly.