Proqr Therapeutics B.V. (PRQR)

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ProQR's sepofarsen shows promise in blindness study
Apr. 13, 2021 4:34 PM ETProQR Therapeutics N.V. (PRQR)By: Aakash Babu, SA News Editor
ProQR Therapeutics (NASDAQ:PRQR) highlights case study describing durable vision improvement after treatment with sepofarsen in a clinical trial for CEP290 mediated Leber congenital amaurosis 10 (LCA10), in the scientific journal Nature Medicine.
Sepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation in the CEP290 gene.
Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children.
"Treatment with sepofarsen resulted in substantial, concordant and enduring improvement in more than a dozen different subjective and objective measurements of visual function as well as retinal structure," lead author Professor Artur V. Cideciyan said.
Published results highlight a patient who is homozygous for the c.2991+1655A>G mutation in CEP290 and was part of a larger cohort in the Phase 1/2 clinical trial. The patient was studied for 15 months after intravitreal treatment with sepofarsen.
Concordant measures of visual function and retinal structure including visual acuity, light sensitivity and visual fields, reached a substantial efficacy peak near three months after injection and remained better than baseline at 15 months.
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https://seekingalpha.com/news/3681528-proqr-highlights-publication-in-nature-medicine-for-sepofarsen-in-blindness-study