Wave Life Sciences announced plans to design and advance stereopure oligonucleotide therapeutics for the potential treatment of rare, inherited eye diseases. Wave’s research in ophthalmology will initially focus on the following four inherited retinal diseases which commonly lead to progressive vision loss typically starting in childhood or adolescence: retinitis pigmentosa due to a P23H mutation in the RHO gene, Stargardt disease, Usher syndrome type 2A and Leber congenital amaurosis 10. The company expects to announce its first ophthalmology development candidate in the second half of 2019. Wave’s decision to expand its therapeutic pipeline into ophthalmology is supported by its data presented at the 14th Annual Meeting of the Oligonucleotide Therapeutics Society. The data demonstrate that a single intravitreal injection of stereopure oligonucleotide in the eye of non-human primates resulted in greater than 95% knockdown of a target RNA in the retina for at least four months. Based on these data, the company is working to design development candidates that could achieve a therapeutic effect with only two doses per year. Wave’s research will assess four genetic targets, RHO P23H, USH2A, ABCA4 and CEP290 to address four rare, inherited retinal diseases. The company estimates that approximately 10,000 U.S. patients could potentially be treated by Wave’s approach to addressing these four diseases.
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