Gene therapy for rare eye disease shows sustained results in long-term follow-up study

Patients with Leber hereditary optic neuropathy (LHON) treated with a single injection gene therapy experienced sustained results 3 years later, according to long-term follow-up study.

A total of 61 patients who had previously participated in the RESCUE and REVERSE Phase 3 pivotal trials where they were treated with Lumevoq in one eye and with sham injection in the other, were included. At 2 years after treatment, participants had had gained an average of  +18.8 and +17.3 letters equivalent relative to the low point of their visual acuity in their treatment-treated eyes and in their sham-treated eyes, respectively. Three years after treatment the bilateral benefit continued with a mean improvement against nadir of +20.5 letters equivalent and sham-treated eyes demonstrating a mean improvement of +19.4 letters equivalent.

“CLIN06 demonstrates that the clinical improvement seen in prior LUMEVOQ studies is real and is maintained for 3 years post treatment,” said Dr. Mark L. Moster, Neuro-Ophthalmology, Wills Eye Hospital, Professor of Neurology and Ophthalmology at Thomas Jefferson University, Philadelphia, PA, and Principal Investigator in the RESCUE, REVERSE and CLIN06 trials, in a press release. “These results are far better than the natural history of LHON and provide further hope for improving the lives of our patients with this blinding disease.”

Read the full press release here.