Jinwon Life Sciences VGXI, Successful Test Production of Plasmid DNA for Chinese Neurophth Neurodegenerative Disease Gene Therapy

Jinwon Life Sciences announced on the 15th that its subsidiary VGXI, a nucleic acid-based biopharmaceutical contract development and manufacturing organization (CDMO), has successfully completed the pilot production of plasmid DNA used as the manufacturing raw material for the adeno-associated virus (AAV)-based ophthalmic disease gene therapy candidate ‘NFS-01 (NR082, rAAV-ND4)’ developed by China’s Neurophth.

Neurophth, a leading Chinese ophthalmic gene therapy company, developed the core product ‘NFS-01,’ which is the first domestically developed AAV-based gene therapy in China to receive orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Additionally, ‘NFS-01’ received Investigational New Drug (IND) approvals from the China National Medical Products Administration (NMPA) in March 2021 and from the U.S. FDA in January 2022. Designated as a Breakthrough Therapy (BTD) in China in July 2022, ‘NFS-01’ completed patient enrollment for its Phase 3 clinical trial in China this February, recently announced positive data from its Phase 1/2 clinical trial in China, and completed the first patient dosing in the global Phase 1/2 clinical trial conducted in the U.S.

Neurophth, located in Wuhan, China, and San Diego, California, U.S., has selected VGXI as its partner for continuous clinical sample production of ‘NFS-01’ and expansion of pharmaceutical manufacturing based in the U.S., collaborating closely since 2020.

Leveraging its technological capabilities, VGXI plays a pivotal role in producing the three essential plasmid DNAs for ‘NFS-01.’ VGXI has also successfully completed pilot production under rigorous quality control supervision through the technology transfer stage and has finalized smooth preparations for master cell bank (MCB) creation and large-scale plasmid DNA production in compliance with Good Manufacturing Practice (GMP) standards.

Youngkeun Park, CEO of Jinwon Life Sciences, stated, “Through trust-based collaboration with Neurophth, we have confirmed the excellence of our plasmid DNA production technology by successfully completing pilot production. We are very pleased to have laid the foundation for accelerating clinical trials and large-scale commercial production of the AAV-based gene therapy ‘NFS-01.’”

Bose Kalampanay, Chief Technology Officer (CTO) of Neurophth, said, “Our clinical trials for new drug development are progressing smoothly, and we look forward to continued collaboration with VGXI to improve the quality of life for patients worldwide suffering from rare ophthalmic diseases with high unmet medical needs through the successful development of this therapy.”

This content was produced with the assistance of AI translation services.