Gene Therapy Promising for Reversal of Hereditary Vision Loss

An unapproved gene therapy for Leber hereditary optic neuropathy (LHON) led to a marked improvement in the eyesight of patients with a severe, progressive form of the disease who received the therapy as part of an early access program.

Results of a study of more than 60 patients who received lenadogene nolparvovec as a unilateral or bilateral intravitreal injection showed that at 2-year follow-up, 60% had experienced a clinically relevant improvement in the number of letters they could read on a visual acuity chart.

The results, said study presenter Chiara La Morgia, MD, PhD, IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy, confirm in a "real-life setting" the efficacy and safety of the treatment as previously shown in clinical trials.

The findings were presented at the Congress of the European Academy of Neurology (EAN) 2023.

Source: MEDspace