Wave Life Sciences developing therapeutics for rare genetic eye diseases

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Wave Life Sciences is planning 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 inherited retinal diseases which commonly lead to progressive vision loss typically starting in childhood or adolescence.

These diseases are: retinitis pigmentosa due to a P23H mutation in the RHO gene, Stargardt disease, Usher syndrome type 2A and Leber congenital amaurosis 10.

In a company update, Wave said it expects to announce its first ophthalmology development candidate in the second half of 2019.

“We have long believed that oligonucleotides have the potential to be particularly effective and durable in the eye and are energized by our latest research that provides additional validation of our precisely designed stereopure oligonucleotides,” said Paul Bolno, President and CEO of Wave Life Sciences.

“Our aim is to move quickly to develop long-acting, intravitreally injected, disease-modifying therapies to address the enormous need across a spectrum of rare, genetically-defined eye diseases.”

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 US patients could potentially be treated by its approach to addressing these four diseases.