Development of microRNA eyedrop to treat nearsightedness
Kaohsiung medical university
本團隊發現微小RNA328過度表現會抑制數個與近視相關的基因，導致近視形成。我們利用干擾性RNA來中和掉過多的微小RNA328，使近視相關的基因表現恢復正常，用來治療近視。於老鼠及兔子近視實驗中以點眼藥水的方式投與，藥物療效令人十分滿意，且沒有觀察到副作用，因此我們的發明有成為新藥的潛力。計畫經由SI2C選題，獲得科技部育苗計畫補助。目前已找出最佳衍生物，其具備安全性高, 製作成本低的優點。準備在2015年8月完成申請Provisional 申請。台灣的市場預計一年約新台幣224億元。未來將推廣至中國大陸、日本及韓國等近視盛行國家，預估市場超過千億。
Myopia (also known as nearsightedness) is a very common eye condition worldwide. Its prevalence is highest in Asians, especially in Chinese descents. In Taiwan, 85% of the high school children have myopia compared to 30% in the USA or European countries. Although USA doctors may not consider myopia a disease, high myopia (defined as -6 diopter or worse) is a well-known risk for several eye diseases including retinal detachment, macular degeneration, glaucoma, cataract and even blindness. Therefore, to prevent myopia progression or to treat myopia can significantly reduce the medical expense and improve quality of life.
The eyeball becomes more elongated in the myopic eye than the normal eye, which is considered a pathological change that is highly corrected with future complications. The treatment of myopia can be divided into two categories – one is to correct refraction without preventing the pathological changes in the eyeball, and the other is to prevent deterioration of refraction as well as to prevent pathological changes. Currently, only mydriasis has been claimed to have mild preventive effect on the progression of myopia and may reduce the abnormal elongation of eyeball. However, mydriasis dilates the pupil, which leads more UV light to penetrate the eyes and damage retina. Furthermore, the dilated pupils makes reading difficult and interfere school learning. Although several methods can be used to correct refraction such as eyeglasses, contact lens, LASIK, othorK contact lens etc, none of which prevent the eyeball from pathological changes. Therefore, there is an unmet medical need to find a better medical/intervention to treat myopia.
Our team first ever demonstrated that microRNA can play a role in myopia development. Furthermore, we use both animal and cellular models to show that over-expression of microRNA-328 can be a risk factor for myopia. microRNA-328 regulates several myopia related genes including PAX6, COL1A1, FMOD etc. Our recent finding further showed that microRNA-328 can suppress scleral stem cells to cause myopia development. Based on these novel mechanisms, we developed microRNA-328 anti-sense oligonucleotides and identified the best candidate to treat myopia. The candidate can be made as eyedrop. In our mouse and rabbit myopia models, we have shown the safety of our candidate, and its efficacy is better than the current mydriasis.
Our project has been selected by SI2C for new drug development and now is fully funded by the Ministry of Science and Technology (MOST) to support all preclinical studies till IND filing. We plan to file provisional patent application in August 2015 and file IND by the end of 2017. Since the current candidate can be synthesized by non-expensive way and materials, we expect the cost to make this eyedrop is low. The estimate market size in Taiwan 22.4 billion NTD per year and the revenue can be much higher when we sale it in mainland China, Japan, and other high prevalent Asian countries.