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WUHAN, China and SAN DIEGO, March 7, 2023 /PRNewswire/ — Matthias Szabo Zarb, a Maltese university student, was diagnosed with Leber hereditary optic neuropathy caused by the ND1 mutation (ND1-LHON) two years ago, which it caused a rapid deterioration of his visual acuity and inability to perform normal activities. Through the National Alliance for Rare Disease Support, he learned about NFS-02, a gene therapy drug targeting ND1-LHON developed by the Chinese ophthalmic gene therapy company, Neurophth. Following a recommendation from the Chinese Embassy in Malta, Michelle Muscat, President of the Maltese National Alliance for Rare Disease Support, contacted Neurophth. Neurophth decided to donate NFS-02 and recommended Dr. Yong Zhang, Director of Taihe Hospital Ophthalmology Center, as the treating physician. Upon his arrival, the patient received free accelerated genetic testing supported by Neurophth. On March 4, Neurophth representatives donated NFS-02 to the Maltese patient at Taihe Hospital. Notably, Professor Zhang Yong performed the surgery for the patient on the same day.

The Chinese Embassy in Malta expressed its appreciation to Neurophth for its support in treating international patients. They praised the company for donating medicines to Matthias Szabo Zarb and his family, as well as fostering closer ties between China and Malta. The collaboration has received a great deal of attention from the Maltese government. The National Alliance for Support of Rare Diseases organized a press conference where the speaker of the Maltese parliament gave a speech. Former Maltese Prime Minister Muscat and his wife also expressed their gratitude to the embassy.

In 2017, Professor Li Bin, former Head of the Department of Ophthalmology, Tongji Hospital, Huazhong University of Science and Technology, launched the world’s largest sample size gene therapy clinical trial for 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 obvious serious adverse events have been found. NFS-02 is Neurophth’s second gene therapy under development for the treatment of mtND1-mediated LHON. After six years of development, the company’s technology platform has become more advanced. NFS-02 has been used to treat several patients in the Investigator Initiated Clinical Trial (IIT) in China and received Orphan Drug Designation and IND approval from the US FDA.

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