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At the beginning of the New Year 2024, Neurophth Biotechnology Co., Ltd. and Sinopharm Group Co., Ltd. jointly announced,Successfully completed the innovative gene therapy drug for Leber's hereditary optic neuropathy — Nurovida®(Validation of Winter Transportation for Eperisone Hydrochloride Ophthalmic Injection).In this snowy winter, Neurophth®The transport vehicle and test samples for the first transport validation in winter arrived as scheduled, successfully reaching the First Affiliated Eye Hospital of Harbin Medical University.
Gene therapy introduces exogenous normal genes into target cells to correct or compensate for diseases caused by defective and abnormal genes, thereby achieving therapeutic purposes. The eye is a closed sphere, and injecting drugs locally in the eyeball has minimal systemic impact, making it suitable for gene therapy. In addition, there are more than 220 genes related to ophthalmology, and any mutation in these genes may cause vision loss and blindness.The world's first drug for treating ophthalmic genetic defects, LUXTURNA®, was developed by Spark Therapeutics, a U.S.-based company, and is used to treat Leber congenital amaurosis (LCA2, RPE65 gene mutation). It was approved for marketing by the U.S. FDA in December 2017. This is considered an important milestone in human gene therapy. Gene therapy, which uses safe viral vectors to deliver nucleic acid drugs to correct genetic abnormalities or express normal gene products, is one of the most promising strategies for treating blinding ophthalmic diseases.Neurophth, as the first ophthalmic gene therapy company in China, is committed to the research and development and industrialization of gene therapy drugs using adeno-associated virus as a vector, exploring and developing new therapies for patients with ophthalmic diseases worldwide.LeberLeber's Hereditary Optic Neuropathy (LHON) is a mitochondrial disease that predominantly affects young men, leading to bilateral vision loss and eventual blindness. Currently, there are no effective treatments or cures available in clinical practice.Neuroptima®(NFS-01) has been validated multiple times for its safety and efficacy. It will be the first AAV gene therapy product in China to complete Phase III clinical trials and is expected to become the world's first innovative drug (First in class) for LHON. To ensure the smooth market launch of the product, Neurophth has actively initiated Nuroviga.®Transport validation. Since gene therapy drugs require ultra-low temperature transportation and storage, this winter transport validation aims to ensure Neurophth.®In extreme environments, stable temperature conditions can be maintained throughout the entire transportation process, whether at the origin, transfer station, or destination, ensuring drug quality.This winter transportation validation took Harbin as the destination. With the support and collaboration of the strategic partner Sinopharm Group Co., Ltd., it lasted for 8 days from January 23 to January 30. Neurophth®The transport vehicle and test samples departed from the Suzhou factory, arrived in Harbin, and safely returned to the Suzhou factory. The validation covered the farthest transportation distance of approximately 2,500 kilometers, the coldest environmental temperature dropping to -20°C, and the longest transportation time exceeding 96 hours. The quality of the drugs was ensured throughout the entire transportation process. The transportation conditions were designed with full consideration of the "most extreme conditions" possible in actual shipments, for Neurophth.®is fully prepared for commercialization.There are currently no commercial standards in China for the transportation and storage of gene therapy drugs, and there are still technical gaps that need to be addressed. The key issue is how to ensure both the safety and efficiency of drug transportation. However, the collaboration between Neurophth and Sinopharm Group Co., Ltd. has meticulously designed solutions for ultra-low temperature storage, transportation, and real-time monitoring, successfully completing validation. This undoubtedly sets an industry benchmark for the logistics management of ophthalmic gene therapy drugs.Dr. Yang Cao, Vice President of Quality Department, Neurophth"Quality is the lifeline of a product, especially for products with high technological content and stringent transportation and storage requirements like AAV gene therapy. Quality assurance is of utmost importance. During the winter transportation validation, our quality team, along with our partner Sinopharm Group Co., Ltd., demonstrated a rigorous and meticulous work ethic, ensuring the stability and safety of the product under extreme conditions. This achievement not only reflects the robustness of our quality management system but also underscores the company's strong sense of responsibility towards customers, the market, and public health. I hope the team will continue to uphold the principle of 'quality first,' continuously optimize processes, strictly control every step, and further enhance the company's reputation for quality."Dr. Zhengbin Li, Vice President of Commercial Department, NeurophthRepresents: "Neurophth"®Is the first AAV gene therapy drug in China about to complete a Phase III registrational clinical trial. This time, Neurophth and Sinopharm Group Co., Ltd. have worked closely together for Neuroviga.®"The 'last kilometer' has been fully prepared to ensure the products can be safely delivered to doctors and patients."With the rapid development of China's gene therapy field, more gene therapy products will gradually enter the commercialization stage. This time, Neurophth®The winter validation has opened the precedent for the commercial transportation and storage of AAV gene therapy drugs in China, serving as a benchmark and leading the Chinese gene therapy industry towards continuous development in standardization and regulation.
Neurophth is a leading gene therapy company in China focused on ophthalmic diseases, with subsidiaries in Wuhan, Suzhou, Shanghai, and San Diego, USA. It is committed to developing gene therapies for hereditary diseases for patients worldwide. The trial data from investigator-initiated retinal gene therapy studies have successfully validated the AAV platform we use, and the results have been published.Nature-Scientific Report、OphthalmologyAndEbioMedicineAbove. Our core product NR082 is designed to treatND4Mediated Leber's Hereditary Optic Neuropathy (ND4-LHON) has been granted Orphan Drug Designation (ODD) by the U.S. FDA and the European Medicines Agency (EMA). It is the first gene therapy new drug developed in China to have received Investigational New Drug (IND) approval from both the China NMPA and the U.S. FDA. The Phase III clinical trial in China has completed patient enrollment and dosing, and the first patient has been enrolled and dosed in the U.S. Phase I/II trial. The company's second U.S. ODD drug, NFS-02, has completed the enrollment and dosing of the first patient in the China-U.S. international multicenter Phase I/II clinical trial.The company's third gene therapy new drug, targetingADOATheNFS-05Approved for clinical trials in Australia.The company's pipeline also includes preclinical candidates for optical neuroprotection and vascular retinopathy.R&D Pipeline
Reference: Neurophth
