On November 30, 2023, Gene Vector Biotechnology Co., Ltd. in Chengdu, China, received approval from the National Medical Products Administration’s Center for Drug Evaluation (CDE) for the clinical trial of JWK001, a Class I gene therapy drug for treating neovascular age-related macular degeneration (nAMD). nAMD, a severe eye disease leading to blindness, is characterized by new blood vessel formation in the macula.
JWK001 is a significant advancement in AAV gene therapy, using a two-plasmid packaging system that is more efficient and cost-effective than the traditional three-plasmid approach. This innovation enhances patient accessibility and is effective for patients unresponsive to single-target treatments. JWK001 introduces an anti-VEGF protein into retinal cells, avoiding the risks associated with frequent traditional nAMD treatments. The initial patient, requiring an average of 10.18 anti-VEGF injections annually, experienced no need for additional treatments for 20 weeks post-JWK001 therapy, and showed considerable improvement in best-corrected visual acuity (BCVA). Professor Yang Yang, CEO and co-founder of Gene Vector Biotechnology, highlighted JWK001’s practicality, requiring only a single dose and showing promising safety and tolerance in early studies.
Gene Vector has developed six AAV gene therapy drugs, including JWK001, which have received ethical approvals and are currently undergoing Investigator Initiated Trials (IIT) at West China Hospital of Sichuan University, covering various medical areas like ophthalmology, genetic metabolism, and neuromuscular diseases.
PackGene Biotech provides analytical testing services for Gene Vector Biotech’s JWK001 IND pipeline, which expedites the clinical trial application process. These services encompass a range of activities including method development, validation, release testing, stability testing, and characterization studies. Committed to making gene therapy both accessible and affordable, PackGene Biotech continually advances its vector process and technology development. This dedication not only aids in drug innovation but also plays a significant role in expanding the application of gene therapy.
To learn more about PackGene’s AAV manufacture and AAV Analytical services, click here.
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
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