Genevector’s JWK002 AAV Gene Therapy for X-Linked Retinoschisis Receives NMPA IND Approval

Genevector Co., Ltd. announced today that its innovative Class I gene therapy, JWK002 Injection, has received Investigational New Drug (IND) approval from China’s National Medical Products Administration (NMPA) for the treatment of X-linked retinoschisis (XLRS). This approval clears the path for clinical trials to commence shortly, marking a significant step forward for patients battling this rare genetic blinding disorder.

JWK002 Injection represents a pivotal moment in the landscape of gene therapy in China, as it stands as the first gene therapy drug for XLRS to gain regulatory clinical trial approval in the country. The therapy has already garnered international recognition, having previously received both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA).

Advanced Manufacturing and Key Partnerships Fuel Progress

Genevector’s proprietary “two-plasmid packaging process system” and advanced suspension HEK293 cell packaging technology underpin the robust CMC (Chemistry, Manufacturing, and Controls) process for JWK002.

Crucially, Packgene has been instrumental in supporting Genevector’s advancement. Packgene has delivered comprehensive, one-stop analytical and testing services for the JWK002 IND pipeline. These vital contributions, encompassing method development and validation, release testing, stability studies, and characterization research across all production scales, were key in accelerating the IND application acceptance for JWK002. Packgene emphasizes its commitment to making gene therapies affordable and accessible, continuing to innovate in vector process development and offering end-to-end CMC solutions for drug innovation and regulatory submissions.

Understanding X-Linked Retinoschisis (XLRS)

XLRS is a debilitating X-linked recessive genetic disorder caused by mutations in the retinoschisin 1 (RS1) gene. It manifests primarily in males, typically diagnosed in early childhood, leading to progressive vision loss, visual field defects, foveal schisis, and reduced retinal electrical activity. Patients can experience varying degrees of visual impairment, with severe cases potentially leading to complications like retinal detachment and vitreous hemorrhage. Current management largely involves observation, supportive medications like carbonic anhydrase inhibitors, and treating complications, with no curative clinical treatment presently available.

How JWK002 Aims to Restore Vision

JWK002 Injection is an adeno-associated virus (AAV) gene therapy product independently developed by Genevector. Engineered through systematic design, including targeted serotype screening and optimization of gene expression elements, JWK002 is designed to efficiently restore RS1 protein expression within retinal cells. The ultimate goal is to improve the structural integrity and functional capacity of the retina in affected patients.

Promising early data emerged from prior investigator-initiated trials (IITs) conducted at West China Hospital of Sichuan University. These studies, which involved dosing multiple subjects with over a year of follow-up, revealed significant improvements in Best Corrected Visual Acuity (BCVA), substantial reductions in Central Retinal Thickness (CRT), and the complete closure of retinal schisis cavities in all treated eyes. Additionally, most treated eyes showed improved retinal photosensitivity, offering compelling early evidence of JWK002’s potential therapeutic benefit.