Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy

This study is testing a gene therapy injection (NR082) for Leber's Hereditary Optic Neuropathy (LHON) — a rare genetic eye disease caused by a mutation in the ND4 gene that causes progressive vision loss, often striking young adults. The injection is delivered directly into the eye and aims to restore vision by providing a functional copy of the affected gene. You may be eligible if: - You are between 12 and 75 years old (or 18–75 in Part 1) - You have vision loss in at least one eye due to LHON confirmed by genetic testing (G11778A mutation in the ND4 gene) - Your vision loss started more than 6 months ago but less than 10 years ago - Your pupils can be adequately dilated for eye examinations - You are willing to use contraception for at least 6 months after treatment You may NOT be eligible if: - You have already received ocular gene therapy in either eye - You have had vitrectomy surgery in either eye - You have other eye diseases that could affect visual assessment - You have HIV, syphilis, or active hepatitis B or C infection - You are pregnant or breastfeeding - You are a heavy smoker (more than 20 cigarettes per day) or have a history of drug or alcohol abuse - You have other known causes of optic nerve disease besides LHON Talk to your doctor to see if this trial is right for you.