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 1 innovative gene therapy drug independently developed by Chengdu Jinweike Biotechnology Co., Ltd. (“Genevector”) for the treatment of neovascular age-related macular degeneration (nAMD), was approved by the National Medical Products Administration (NMPA). PackGene Biotech extends its sincere congratulations to Genevector on this important progress.

Academician Wei Yuquan, Founder and Chairman of Genevector, stated: “We are very pleased that the IND application for JWK001 Injection, our first independently developed innovative drug, has been approved. This marks an important milestone. Based on the follow-up results from the IIT studies we have conducted, JWK001 Injection has demonstrated favorable safety and tolerability, with a low clinically 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 the Wenjiang District Government, West China Hospital of Sichuan University, Professor Lu Fang’s ophthalmology team, and all shareholders of Genevector for their strong support.”

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 clinical trial application for the gene therapy drug JWK001 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 JWK001

JWK001 Injection is the first AAV gene therapy drug developed using 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 stable and cost-effective CMC process is expected to offer significant advantages in both clinical and commercial stages, greatly improving patient accessibility.

Based on its independently developed two-plasmid packaging system technology platform, Genevector is advancing multiple AAV gene therapy candidates in IIT studies at West China Hospital of Sichuan University, with indications covering ophthalmic diseases, inherited metabolic diseases, and neuromuscular disorders.

JWK001 Injection utilizes an AAV vector carrying a newly and independently designed anti-VEGF protein expression cassette to enable sustained and efficient expression of anti-VEGF protein in retinal cells. This gene therapy approach may help avoid risks associated with traditional nAMD treatment, 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 biotechnology company dedicated to the development of innovative AAV gene therapy drugs for ophthalmic diseases, neuromuscular disorders, inherited metabolic diseases, and other conditions. Multiple AAV gene therapy products in its pipeline have entered the clinical research stage.

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 150 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.

About PackGene Biotech

PackGene Biotech (PackGene) is a CRO & CTDMO technology company specializing in recombinant adeno-associated virus (rAAV) vector packaging. The company provides fast, cost-effective, high-quality, and scalable one-stop CMC solutions for cell and gene therapy (CGT) companies, supporting recombinant AAV vector drugs from early-stage R&D and preclinical development to clinical trials and regulatory approval.

Since its establishment in 2014, PackGene has become a leader in the AAV vector CRO service field, delivering tens of thousands of batches of customized AAV samples to clients in more than 20 countries, including top global multinational pharmaceutical companies and various research institutions. In the CTDMO service field, PackGene leverages five major technology platforms, including the π-Alpha 293 high-yield AAV production platform and the π-Omega high-yield plasmid production platform, together with a comprehensive quality and analytical system, to provide compliant services for GMP-scale AAV and plasmid manufacturing for pharmaceutical companies.

With the mission of “make gene therapy affordable” and the responsibility of accelerating the launch of innovative gene therapies, PackGene Biotech is committed to becoming a long-term global partner for gene therapy R&D and manufacturing. Built on innovative technologies, PackGene is driven by two core directions—advanced vector production technologies and breakthrough vector tools—to establish an international brand in the CRO and CTDMO fields of gene therapy.