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Week in review: CRISPR start, RP first, 50-year case

By Keng Jin Lee & Kanaga Rajan

A weekly roundup of ophthalmic news from around the web.

Editas Medicine is “making history” with their in vivo CRISPR genome-editing trial, the company’s CEO said during a recent healthcare conference. Enrollment has begun for the early-phase trial that will test a single subretinal injection of EDIT-101 in patients with Leber congenital amaurosis 10. They expect the first patient dosing to take place in the first quarter of 2020. Editas Medicine

The FDA awarded a rare pediatric disease designation to a first-in-class investigational treatment for retinitis pigmentosa (RP), according to an announcement by ProQR. Their RNA-based oligonucleotide therapy targets diseases caused by mutations in the USH2A gene. The company hopes that the intravitreal treatment will address vision loss associated with Usher syndrome type 2 and nonsyndromic retinitis pigmentosa. ProQR


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