Trial sponsor China

Neurophth Therapeutics

Wuhan Neurophth Biotechnology

China-based biotech running the most advanced commercial LHON gene therapy program. GOLD trial (NCT04912843, Phase 1/2/3) and bilateral IVT Phase 3 (NCT07406854) target ND4 mutations. Also pursued NFS-02 for ND1 (NCT05820152, terminated 2024).

Research focus

NR082 gene therapyNFS-02commercial LHON therapeutics

Public profiles

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Relevant project findings

Neurophth is the most commercially advanced LHON gene therapy sponsor globally, with two active Phase 3 trials for NR082. Their competitive landscape against GenSight’s LUMEVOQ makes them a key stakeholder for trial sponsor outreach.

Project outreach status

Not yet contacted

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