An international clinical trial conducted using the Perelman School of Medicine at the University of Pennsylvania helped a patient who was suffering from childhood blindness to gain vision after a single injection of experimental RNA therapy into the patient’s eye.
The potentially innovative research was published in an article in Nature Medicine on April 1st.
During the trial, patients received intraocular injections, shots to the eye, from an antisense oligonucleotide known as sepofarsen, a small RNA molecule that targets the cells in the eye cone – which are responsible for color vision – to increase levels of CEP290 protein and improve the functioning of the retina during the day.
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The potential treatment is aimed at patients suffering from Leber congenital amaurosis (ACL), a rare and genetic eye disease that affects the retina, and who also have a CEP290 mutation.
Usually, ACL patients are blind or have severe visual impairment starting in childhood.
In this study, one of the patients was followed up for 15 months after the single injection. The patient’s vision improved after one month, peaked in two months and continued to improve even 15 months after the single injection.
“Our results set a new standard for what biological improvements are possible,” said Artur Cideciyan, co-author of the study and a research professor of ophthalmology at the Scheie Eye Institute at Penn Medicine.
For the study’s authors, including co-author Samuel Jacobson, the long schedule of improved vision after an injection gives them hope that RNA therapy will have similar impacts on other ciliopathies, or eye genetic mutations with protein defects.
“This work represents a really exciting direction for antisense RNA therapy. It has been 30 years since new drugs appeared using antisense RNA oligonucleotides, even though everyone realized that there was great promise for these treatments ”. said Jacobson.
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