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Posts tagged USH2A

ProQR Receives FDA Authorization to Launch Clinical Trial for USH2A Therapy

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ProQR, a biotech in the Netherlands developing therapies for rare diseases, has received authorization from the US Food and Drug Administration to launch a Phase 1/2 clinical trial for QR-421a, its treatment targeting mutations in exon 13 of the USH2A gene. The mutations cause Usher syndrome type 2A (combined vision and hearing loss) and non-syndromic retinitis pigmentosa (vision loss only) in approximately 16,000 people in the Western World. ProQR plans to begin enrolling patients in the QR-421a Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.
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FFB-CRI Investing $7.5 Million in Emerging Therapy for USH2A

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The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene. FFB-CRI will be investing up to $7.5 million in milestone-based funding to advance the treatment, known as QR-421a, toward a Phase 1/2 clinical trial during 2018. ProQR plans to issue the initial data report for the clinical study in 2019.
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Top Retinal Research Advances for 2017

To view and listen to Ben Shaberman’s presentation of “Top Retinal Research Advances for 2017,” with full slides and audio, click here. The text to the presentation is as follows:

This is Ben Shaberman, director of science communications, at the Foundation Fighting Blindness (FFB), and I’m pleased to present a quick overview of some of the exciting research advances for inherited retinal diseases made during 2017. It has been an exciting year with several promising therapies moving into and through clinical trials.
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ARVO 2015 Highlight: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes

gene editingI just returned from the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), the world’s largest eye-research conference, held this year in Denver. It attracted more than 11,000 scientists and physicians, including many of the 187 retinal researchers funded by the Foundation. The FFB science team and I worked feverishly to learn as much as possible about the latest news from the retinal-research front. It was truly exhilarating—albeit, at times, overwhelming.
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