China's First Gene Therapy for X-Linked Retinoschisis, JWK002 Injection, Receives IND Approval from NMPA

Jun 25, 2025 12:00 CST Updated 12:00

Jinweike Biotechnology

AAV Gene Therapy New Drug Developer

Recently, the Investigational New Drug (IND) application for "JWK002 Injection," a Class I innovative gene therapy drug independently developed by Chengdu Jinweike Biotechnology Co., Ltd. ("Jinweike") for the treatment of X-linked retinoschisis, has been successfully approved by the National Medical Products Administration (NMPA). Clinical trials are expected to commence shortly.As an important strategic partner of Jinweike Biotechnology, PackGene Biotherapeutics expresses sincere congratulations on this significant progress!

JWK002 InjectionIs the first in ChinaForGene Therapy for X-Linked RetinoschisisDrug Registration Clinical Trials. Previously,JWK002AlreadyObtained in the United StatesFDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD)。

The CMC process of JWK002 Injection adopts Jinweike's self-developed "two-plasmid packaging process system." The suspension HEK293 cell packaging technology is stable, easy to scale up, and significantly reduces production costs. It will have very obvious advantages during the clinical and commercial stages, greatly improving patient accessibility.

Paigen BiotechnologyDeeply honored to assist Jinweike Biotechnology in the progress of its AAV gene therapy drugs, providing one-stop analytical testing services for its IND pipeline.Including method development and validation, release testing, stability testing, and characterization studies, providing comprehensive testing and release for products from small-scale to pilot-scale production, accelerating the clinical trial application and acceptance of the gene therapy new drug JWK002 injection!In the future, PackGene Biotech will continue to uphold the mission of "making gene therapy affordable for the common people," constantly advancing the development of vector processes and technologies, providing a one-stop CMC solution for drug innovation, and promoting the application of gene therapy drugs!

About X-linked Retinoschisis

X-linked Retinoschisis (XLRS) is an X-linked recessive genetic disorder caused by mutations in the retinoschisin 1 (RS1) gene. It is characterized by varying degrees of vision loss, visual field defects, foveal schisis, and a decrease in the electroretinogram b-wave. XLRS primarily affects males and is typically diagnosed in early childhood. The severity of visual impairment varies among patients, with severe cases potentially leading to complications such as retinal detachment and vitreous hemorrhage. Clinical management mainly involves follow-up observation, carbonic anhydrase inhibitors, and treatments targeting its complications.There is currently no effective clinical treatment available.

About JWK002Injection

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

In the IIT clinical study previously conducted at West China Hospital of Sichuan University,Have completed dosing for multiple subjects and exceeded1-year follow-up.The results showed that all treated eyes had significant improvement in best-corrected visual acuity (BCVA), significant reduction in central retinal thickness (CRT), complete closure of retinoschisis cavities, and improvement in retinal light sensitivity was observed in the majority of treated eyes.

Fig: After treatment, the patient's retinoschisis cavity returned to normal.

JinWeike BiotechnologyCo-founder,CEOResearcher Yang Yang stated:

"Very pleased that our independently developed JWK002 has entered the registration clinical stage, marking another significant milestone for the company. Based on the ongoing follow-up results from the IIT clinical studies already conducted on JWK002, the drug has demonstrated good safety and tolerability, with a low effective dose. Patients only require a single administration without adjuvant therapy, providing great convenience to patients. Moving forward, our team will accelerate the clinical progress to benefit more patients. Here, we would like to express our special thanks to West China Hospital of Sichuan University and the ophthalmology team led by Professor Lu Fang, as well as Jinweike's shareholders for their strong support of the company."

About Jinweike

Jinweike Biotechnology is a biotech company dedicated to the development of new AAV gene therapies for ophthalmic diseases, neuromuscular disorders, and inherited metabolic conditions.Currently, two AAV gene therapy products in the research pipeline have entered the registration clinical stage, and several other pipelines have entered the IIT clinical research stage.

Under the leadership of Academician Yuquan Wei and Researcher Yang Yang, Jinweike Biotechnology has established a 3,000-square-meter R&D and production base that integrates AAV gene drug research and development, pilot production, and quality control. It has formed a highly integrated and comprehensive AAV gene therapy "technology chain." The company’s R&D capabilities for gene therapy drugs and its AAV preparation technology are at the leading level in China. The AAV gene therapy products prepared by the company have completed clinical studies involving more than 100 patients, demonstrating excellent safety and efficacy data.

The core management team of Jinweike Biotechnology comes from well-known pharmaceutical companies, research institutions, and universities both in China and abroad. The background of the core technical team covers AAV vector design, pilot-scale production, quality research, and medical clinical trials, endowing Jinweike with integrated operational capabilities ranging from the original design of gene therapy products, scalable pilot manufacturing, to medical and clinical research.

Jinweike Biotechnology will leverage its robust technical platform to collaborate with leading industry partners both in China and internationally, integrating resources to enhance the accessibility of AAV gene therapy drugs, striving to benefit a wide range of patients more rapidly.

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