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Intriguing interim results
May 2020
SHARING OPTIONS:

LEIDEN, the Netherlands & CAMBRIDGE, Mass.—A three-month interim analysis of ProQR Therapeutics N.V.’s Phase 1/2 Stellar trial yielded positive results in adults with Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A exon 13 mutations. QR-421a has been generally well tolerated with no serious adverse events so far. Patients in the “sham” cohort showed no consistent pattern of response above the “noise” cohort, but two of the eight patients who received QR-421a saw improvement. One of the four treated patients in the low-dose group responded to treatment, with the benefit maintained for six months or more. The patient improved on the full field stimulus threshold test (FST), dark adapted chromatic (DAC) perimetry and optical coherence tomography assessment of photoreceptor Ellipsoid Zone. Another patient responded in the mid-dose group, seeing improvement in FST, DAC and BCVA (a seven-letter improvement from baseline).

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