Neurophth Completes Enrollment for Opvika® LHON Gene Therapy Trial in the U.S.

20 February 2024 | Tuesday | News

The trial aims to advance treatment for Leber hereditary optic neuropathy with pioneering gene therapy, marking a significant stride in genetic eye disease research.
Image Source : Public Domain

Image Source : Public Domain

Neurophth Therapeutics, Inc. ("Neurophth"), a pioneer in gene therapies, today announced the successful completion of patient enrollment for its Phase I/II clinical trial of Opvika® (Esonadogene Imvoparvovec) in the United States. This trial targets the treatment of Leber hereditary optic neuropathy (LHON) caused by the ND4 mutation (ND4-LHON), a genetic condition that leads to acute or subacute vision loss primarily in young adults.

The Phase I/II study is a single-arm, multi-center evaluation of the safety and efficacy of NR082, Neurophth’s promising gene therapy candidate for LHON patients with ND4 mutations. Following the U.S. Food and Drug Administration’s (FDA) approval of the Investigational New Drug (IND) application on January 18, 2022, the first patient received a dose in June 2023, marking a significant milestone in the pursuit of innovative treatments for genetic eye diseases.

Professor Li Bin, Founder, Chairman, and CEO of Neurophth, expressed deep gratitude for the commitment shown by patients, families, and researchers throughout the enrollment process. He highlighted the company’s ambition to extend China’s medical advancements to the global stage, emphasizing the goal of delivering cutting-edge gene therapies to patients worldwide.

The study’s principal investigator, Prof. Yaping Joyce Liao of the Stanford Health Care (SHC) - Byers Eye Institute, shared her enthusiasm about reaching this critical stage of the trial. Prof. Liao stressed the urgent need for new treatments among the LHON community and expressed optimism about the potential impact of Opvika® on the lives of those affected by this debilitating condition.

With patient enrollment now complete, the focus shifts to the next phases of the trial, where researchers and the global LHON community keenly anticipate the outcomes. This study represents a hopeful step forward in the quest for effective treatments for LHON, potentially setting a new standard in gene therapy for genetic eye diseases.

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