A new study of an experimental RNA therapy revealed that, for one patient with congenital blindness, it’s given that patient the gift of sight.
The news comes courtesy of Interesting Engineering, who reported on a study from Nature discussing the use of antisense oligonucleotide (AON) sepofarsen in patients with a form of congenital blindness.
As the article explained, “The treatment was designed for people pre-diagnosed with Leber congenital amaurosis (LCA) — which is an eye disorder that typically affects the retina — who also have a CEP290 mutation, a gene commonly associated in patients with the disease. People who have this type of LCA generally suffer severe visual impairment from infancy onward.”
Research Professor of Ophthalmology Artur V. Cideciyan, reporting on the study, noted, “Our results set a new standard of what biological improvements are possible” with the treatment.
The study monitored 10 patients who received the experimental RNA therapy once per three months, which caused an increase in vision capability for 10 patients. As the article explained, “The eleventh patient — whose treatment was explained in the new paper of the same journal, was given just one injection, and then examined over a 15-month timeframe. Before treatment, the patient suffered from reduced visual acuity, small visual fields, and no night vision. But after the shot, the scientists decided not to add quarterly maintenance doses — since doing so could cause cataracts.”
That patient, monitored over the same 15-month period, experienced improved vision after just a month, and while the effects of the treatment peaked at the two-month mark, it still has positive effects on the patient’s vision at the end of the monitoring period.
“This work represents a really exciting direction for RNA antisense therapy,” said Samuel G. Jacobson, a co-author on the research, adding that it’s been 30 years since there has been new drugs of this kind, “even though everybody realized that there was great promise for these treatments.”