Neurophth Therapeutics, Inc.
Wuhan, China and San Diego, March 7, 2023 (Reuters) /PRNewswire/
Matthias Szabo Zarb, a Maltese student, was diagnosed two years ago with Leber’s hereditary optic neuropathy caused by the ND1-Mutation (ND1-LHON) is caused. This led to a rapid deterioration in his visual acuity and his inability to engage in normal activities. He learned about NFS-02, a gene therapy drug, from the National Alliance for Rare Diseases Support ND1 -LHON and is being developed by Chinese gene therapy company Neurophth. On the recommendation of the Chinese Embassy in Malta, Ms. Michelle Muscat, Chair of the Malta National Alliance for Rare Diseases Support, contacted Neurophth. Neurophth decided to donate NFS-02 and recommended Dr. Yong Zhang, director of the Ophthalmology Center at Taihe Hospital, as the attending physician. Upon arrival, the patient received free rapid genetic tests sponsored by Neurophth. On March 4th, representatives of Neurophth donated NFS-02 to the Maltese patient in Taihe Hospital. Professor Zhang Yong performed the surgery on the patient the same day.
The Chinese Embassy in Malta thanked Neurophth for its support in treating international patients. She commended the company for donating the drug to Mr. Matthias Szabo Zarb and his family and for promoting closer ties between China and Malta. The cooperation has attracted a great deal of attention from the Maltese government. The National Alliance for Rare Diseases Support organized a press conference at which the Speaker of the Maltese Parliament made a speech. Former Maltese Prime Minister Muscat and his wife also offered their appreciation to the embassy.
In 2017, Professor Li Bin, former director of the Department of Ophthalmology at Tongji Hospital, Huazhong University of Science and Technology, launched the world‘s largest clinical trial of gene therapy ND4-LHON. The study included 149 patients from China and 10 patients from Argentina. The results of the 12-month follow-up showed that NFS-01 has a remarkable curative effect and no apparent serious adverse events occurred. NFS-02 is Neurophth’s second gene therapy being developed for the treatment of mtND1-mediated LHON. After six years of development, the company’s technology platform is becoming more and more advanced. NFS-02 has been used to treat multiple patients in the clinical trial (IIT) in China and received orphan drug designation and IND approval from the US FDA.
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Original content from: Neurophth Therapeutics, Inc., transmitted by news aktuell