High myopia increases risk for severe complications such as macular degeneration, retinal detachment, which can lead to blindness. Given an extremely high prevalence of myopia in Asia, we aim to develop a novel and effective eyedrop to prevent myopia progression.
ORAL PRESENTATION: 1:30PM
We discovered that a microRNA is over-expressed in myopic eyes, and this microRNA regulates expression of myopia-related genes. An anti-sense oligonucleotides was designed to neutralize over-expressed microRNA. Cellular and animal studies were conducted to test the effect of this oligonucleotide.
The oligonucleotide was first demonstrated to effectively neutralize excess microRNA in the cells. In addition, this oligonucleotide does not cause apoptosis or inflammation in the ocular cells. Using monocular form-deprivation to induce myopia in mice and rabbits, we observed that the myopic eye is longer than the fellow eye in the same animal. This oligonucleotide was prepared in the eyedrop form to treat myopic eyes, while we used 1% atropine and vehicle as the positive and negative controls, respectively. The dosing frequency was once per week for mice and every other day for rabbits. Our in-house data on mouse eyes showed that our eyedrop can reduce eyeball elongation by 0.055 mm, while 1% atropine reduces 0.039 mm and negative control increases 0.01 mm. Notably, 1% atropine is 4-fold more effective than clinically used 0.1% atropine. The eyeball length is approximately 3 mm in a mouse. The result was also replicated by an independent CRO company. For the rabbit study, we demonstrated the dose-dependent reduction of eyeball length in the myopic eyes. The radio-labeled study showed that the oligonucleotide eyedrop can penetrate cornea and also flow along the sclera. The PK study showed that 1/500 oligonucleotide can be detected in the circulation when the oligonucleotide is applied to the eyes. The initial studies did not reveal any side effects.
Atropine is the only effective drug to treat myopia currently. However, its side effects (pupil dilatation and photophobia) lead to low compliance and a concern of UV-induced damage. There is an unmet medical need to develop a more effective and safer treatment. Our product has shown effectiveness, convenient and safety, and it has a potential to become a novel and effective anti-myopia eyedrop. We are looking for partners to co-develop this eyedrop.