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 I innovative gene therapy drug independently developed by Chengdu GeneVector Biotechnology Co., Ltd. (“GeneVector”) for the treatment of X-linked retinoschisis (XLRS), was successfully approved by the National Medical Products Administration (NMPA). Clinical trials are expected to be initiated in the near future. As an important strategic partner of GeneVector, PackGene extends its heartfelt congratulations on this significant progress.

JWK002 Injection represents China’s first registered clinical trial of a gene therapy drug for X-linked retinoschisis. Previously, JWK002 had been granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the U.S. 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 easy to scale up, significantly reducing production costs. This will provide clear advantages in both the clinical and commercialization stages and greatly improve patient accessibility.

PackGene is honored to support the progress of GeneVector’s AAV gene therapy drug. 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 services for products from small-scale to pilot-scale production, accelerating the acceptance of the clinical trial application for the innovative gene therapy drug JWK002 Injection. In the future, PackGene will continue to uphold its mission of “making gene therapy affordable for ordinary people,” further deepen the development of vector processes and technologies, provide one-stop CMC solutions for drug innovation, and support the filing 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 consists of follow-up observation, medications such as carbonic anhydrase inhibitors, and treatment of complications. There is currently no effective clinical treatment 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 the expression of RS1 protein in retinal cells, thereby improving retinal structure and function in patients.

In the previous investigator-initiated trial (IIT) conducted at West China Hospital of Sichuan University, multiple subjects had completed administration and more than one year of follow-up. Results showed that all treated eyes demonstrated significant improvement in best-corrected visual acuity (BCVA), significant reduction in central retinal thickness (CRT), complete closure of the retinal schisis cavities, and improvement in retinal photosensitivity in most treated eyes.

Figure: After treatment, the patient’s retinal schisis cavities returned to normal.

Researcher Yang Yang, co-founder and CEO of GeneVector, said:

“We are very pleased that our independently developed JWK002 has entered the registered clinical stage, marking another important milestone for the company. Based on the continuous follow-up results from the IIT clinical study already conducted for JWK002, the drug has demonstrated good safety and tolerability, a low effective dose, and requires only a single administration without adjunctive therapy, bringing great convenience to patients. Going forward, our team will accelerate clinical development so that more patients can benefit. We would especially like to thank West China Hospital of Sichuan University, Professor Fang Lu’s ophthalmology team, and GeneVector’s shareholders for their strong support of the company.”

About GeneVector

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

Under the leadership of Academician Yuquan Wei and Researcher Yang Yang, GeneVector has established a 3,000-square-meter R&D and production facility integrating AAV gene drug research and 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 development and AAV manufacturing technology. AAV gene therapy products manufactured by the company have completed clinical studies in more than 100 patients, demonstrating excellent safety and efficacy data.

GeneVector’s core management team comes from renowned 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 achieve integrated operational capabilities spanning the source design of gene therapy products, scaled pilot manufacturing, and medical and clinical research.

Leveraging its robust technology platform, GeneVector will collaborate with leading industry partners in China and abroad, integrate resources, improve the accessibility of AAV gene therapy drugs, and strive to benefit patients more quickly.