ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, has dosed the first patient in the Phase 1\/2 STELLAR clinical trial for QR-421a in patients with Usher syndrome type 2 or non-syndromic retinitis pigmentosa (RP). Interim data from the study are expected to be announced mid-2019.
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“There are no effective treatments for most inherited retinal diseases, including Usher syndrome, and blindness often results,\u201d said David G. Birch, Ph.D., Principal Investigator of STELLAR and ScientificDirector of the Retina Foundation of the Southwest in Dallas, Texas. \u201cThe STELLAR study is one of the first studies of its kind exploring the impact of ProQR\u2019s RNA therapies on patients with Usher syndrome due to an Exon 13 mutation. The STELLAR trial will explore whether QR-421a (ProQR\u2019s RNA therapy) can slow disease progression or even reverse it. Treatments such as this, that target the underlying cause of a disorder, have the potential to give new hope to patients and their families that life-changing therapies could be available in the near future.\u201d<\/p>\n\n\n\n
\u201cUsher syndrome is a devastating disease, so we are pleased to advance QR-421a into the clinic with the goal to make a difference for these patients, similar to what we have observed with early but promising data for sepofarsen in patients with LCA10,\u201d said Daniel A. de Boer, chief executive officer of ProQR. \u201cWe are committed to rapidly advancing our promising RNA therapies for inherited retinal diseases and we believe our platform of generating targeted RNA therapies with long retinal half-lives and the ability to reach both central and peripheral retina, we will be able to target many of these diseases in the coming years.\u201d<\/p>\n\n\n\n
Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the USH2A gene targeted by QR-421a cause vision loss in approximately 16,000 individuals in the Western world.<\/p>\n\n\n\n