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Posts tagged QR-421a

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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