Warm congratulations to GeneVector on the IND approval of its AAV gene therapy drug, the first to adopt a two-plasmid packaging system

On February 22, 2024, the Investigational New Drug (IND) application for JWK001 Injection, a Class I innovative gene therapy drug independently developed by Chengdu GeneVector Biotechnology Co., Ltd. (“GeneVector”) for neovascular age-related macular degeneration (nAMD), was successfully approved by the National Medical Products Administration (NMPA). PackGene extends its sincere congratulations to GeneVector on this important progress.

Professor Yuquan Wei, founder and chairman of GeneVector and Academician of the Chinese Academy of Sciences, said: “We are very pleased that the IND application for JWK001 Injection, our first independently developed innovative drug, has been approved. This is an important milestone. Based on the follow-up results from the IIT study we have already conducted, JWK001 Injection has demonstrated good safety and tolerability, a low clinically 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 the Wenjiang District Government, West China Hospital of Sichuan University, Professor Fang Lu’s ophthalmology team, and all shareholders of GeneVector for their strong support.”

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 clinical trial application for the innovative gene therapy drug JWK001 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 JWK001

JWK001 Injection is the first AAV gene therapy drug to adopt a “two-plasmid packaging system.” GeneVector’s independently developed two-plasmid suspension HEK293 cell packaging technology significantly improves AAV packaging efficiency and reduces production costs. GeneVector’s CMC process is stable and cost-effective, providing significant advantages in both the clinical and commercialization stages and greatly improving patient accessibility.

Based on its self-developed “two-plasmid packaging system” technology platform, several AAV gene therapy drugs developed by GeneVector are currently undergoing investigator-initiated trials (IITs) at West China Hospital of Sichuan University, with indications covering ophthalmic diseases, inherited metabolic diseases, and neuromuscular diseases.

JWK001 Injection uses an AAV vector carrying a newly and independently designed anti-VEGF protein expression cassette to achieve sustained and efficient expression of anti-VEGF protein in retinal cells. This gene therapy approach can avoid risks associated with traditional nAMD treatments, such as damage to ocular tissues caused by repeated intravitreal injections and poor patient compliance, with the goal of achieving lifelong efficacy through a single treatment.

About GeneVector

GeneVector is a company dedicated to the development of innovative AAV gene therapy drugs for ophthalmic diseases, neuromuscular diseases, inherited metabolic diseases, and other conditions. Multiple AAV gene therapy products in its pipeline have already entered the clinical research stage.

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 150 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.