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February 7, 2022: ProQR and the Usher Syndrome Coalition hosted a webinar about the newly announced clinical trials, “Sirius” and “Celeste,” for individuals with Usher syndrome type 2A (USH2A).
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QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events — QR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platform — COVID-19 pandemic expected to impact timelines for the pipeline — ProQR anticipates its cash runway will fund operations into H2 2022
LEIDEN, Netherlands & CAMBRIDGE, Mass.,
Read ProQR is developing a novel investigational drug called QR-421a to treat RP in patients that have USH2 due to a mutation in a specific part of the USH2A gene, called exon 13.
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