VGXI, Inc. proudly announces the successful completion of the engineering run for plasmid DNA used in Neurophth's AAV ocular gene therapy.
CONROE, Texas , Nov. 14, 2023 /PRNewswire-PRWeb/ -- VGXI, Inc., a leading contract development and manufacturing organization (CDMO) specializing in nucleic acid biopharmaceuticals, including gene therapies and DNA vaccines, proudly announces the successful completion of the engineering run for plasmid DNA used in Neurophth's AAV ocular gene therapy candidate, NFS-01.
Neurophth, China's leading clinical-stage, in-vivo gene therapy company dedicated to combatting opthalmic diseases, has collaborated closely with VGXI since 2020. VGXI played a pivotal role in the development of NFS-01, by supplying all three essential plasmid constructs to formulate the AAV candidate. Neurophth selected VGXI as a manufacturing partner to expand U.S.-based manufacturing in support of ongoing clinical production. VGXI's achievements include the generation of GMP Master Cell Banks (MCBs) and a seamless transition into Good Manufacturing Practice (GMP) bulk plasmid manufacturing.
NFS-01 is China's first self-developed AAV-based gene therapy to treat Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON), a condition currently lacking an effective treatment or cure. It was granted orphan drug designation by the FDA and EMA. It received China IND clearance from the NMPA in March 2021‥and U.S. IND clearance from the FDA in January 2022. Neurophth has completed patient enrollment for the Phase III clinical trial and announced positive data from the Phase I/II clinical trial in China recently. Meanwhile, the first patient has been dosed in the international multi-region, multi-center Phase I/II clinical trial in the U.S.
In addition to NFS-01, Neurophth successfully administered the first dose of NFS-02 to patients in August 2023 as part of an international multi-region, multi-center Phase I/II clinical trial. This trial aims to treat Leber hereditary optic neuropathy caused by ND1 mutation (ND1-LHON), another condition currently lacking an effective treatment or cure. This product is Neurophth's second gene therapy to enter clinical trials and the only ND1-LHON treatment in development worldwide. The material produced by VGXI will not only be integral to the ongoing clinical trial but will also provide crucial support for late-stage testing and eventual commercialization upon regulatory approval.
The progress made thus far exemplifies the dedication and expertise of both Neuropthth and VGXI in advancing groundbreaking gene therapies.
"We are thrilled to have partnered with Neurophth in their journey towards bringing innovative ocular gene therapies to patients worldwide," stated Young Park, CEO of VGXI. "This successful engineering run is a testament to the collaborative efforts of our teams and the commitment to excellence that drives our work. We look forward to continuing our partnership and remain dedicated to supporting Neurophth as they continue their pursuit of commercial approval for their AAV ocular gene therapy."
"Thank you to VGXI's cutting-edge plasmid DNA platform. Our new drug development and clinical trials are progressing smoothly." said Bose Kalampanayil, CTO of Neurophth. "It is a significant step toward addressing unmet medical needs in ocular diseases. We eagerly anticipate further collaboration in expediting the commercial manufacturing of our gene therapy, delivering safe and effective treatments to patients worldwide."
ABOUT VGXI, INC.
VGXI is a leader in plasmid DNA contract manufacturing, with 20+ years of experience providing high quality products to biomedical researchers worldwide. We support clients that develop DNA vaccines, immunotherapies, cell therapies, and gene therapies. VGXI's manufacturing process and quality controls are coupled to a continuous, low-shear AIRMIX® patented lysis technology, and optimized purification processes that provide highly supercoiled plasmid and exceptional purity. VGXI's portfolio includes high quality plasmid for preclinical research, Highly Documented (HD) plasmid as a critical raw material for GMP viral vector production or pharm/tox studies, cGMP plasmid DNA for clinical through commercial supply, and custom services. VGXI, Inc. is a GeneOne company.‥To learn more, visit‥https://www.vgxii.com.
Neurophth is China' leading in-vivo gene therapy company for ophthalmic diseases. With subsidiaries in China (Wuhan, Shanghai, and Suzhou) and US (San Diego, California), Neurophth, a fully integrated company, is striving to discover and develop genomic medicines for patients suffering from genetic diseases globally. Our validated AAV platform, which has been published in‥Nature - Scientific Reports, Ophthalmology, and EBioMedicine, has successfully delivered proof-of-concept investigator-initiated trials data of 186 subjects with investigational gene therapies in the retina. Our most advanced investigational gene therapy drug candidate, NFS-01 (rAAV2-ND4), in development for the treatment of mtND4-mediated LHON, has been granted orphan drug designation (ODD) by the U.S. FDA and EMA. After the IND clearance by the China NMPA in March 2021‥and the U.S. IND by FDA in January 2022, Neurophth has completed patient enrollment for Phase III clinical trial in China and dosed the patient in the U.S. Phase I/II clinical trial. Recently, our second gene therapy NFS-02, has dosed the patient in China-U.S. Phase I/II clinical trial. The pipeline also includes autosomal dominant optic atrophy, optic neuroprotection, vascular retinopathy, and five other preclinical candidates. To learn more about us and our growing pipeline, visit http://www.neurophth.com.