Our story begins back in 2011 with Dr. Bin Li, a neuro-ophthalmologist of Tongji Medical College of Huazhong University of Science and Technology. Dr. Li conducted the first gene therapy treatment in Leber’s Hereditary Optic Neuropathy (LHON) through an investigator-initiated trial (IIT) of 9 patients.

At 36 months following treatment, 7 of the patients had at least 0.3 logMAR improvement, which is the standard for clinically meaningful improvement in best-corrected visual acuity. We have been able to stay in touch with most of these patients, making this some of the most longitudinal in-human gene therapy data.

Neurophth is founded with a mission to develop genetic therapies for ophthalmic diseases.

Following promising results from the 2011-2012 IIT, Dr. Bin Li’s group conducted a second IIT in 2017-2018 that would go on to include 159 patients. These studies have shown that the gene therapy treatment is well-tolerated and can deliver clinically meaningful visual acuity improvement to patients. Between the two studies.

The President of the Argentina Stargardt association reaches our to Dr. Li on behalf of Argentinian children with LHON. Dr. Li’s group works with Argentinian doctors to have 10 patients receive treatment in China and remain in contact after returning home.

Data from second real-world study continues to be collected for all patients. Of the patients followed up to 12-months so far, treatment has been well-tolerated and a large proportion of patients have clinically significant visual acuity improvement.

Neurophth remains dedicated to developing gene therapy treatment for LHON and other ophthalmic conditions.