… Full field stimulus testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis … phase 1b/2 trial, 11 LCA10 patients received intravitreal sepofarsen . The goal was to evaluate patient response to …
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
ARVO 2021 presentation by Stephen R. Russell from Iowa University on the Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in CEP290 mediated Leber Congenital Amaurosis type 10.
… Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber … Stephen Russel et al. Recognized with best poster award. Sepofarsen had a manageable safety profile and showed …
… ProQR Announces Publication in Nature Medicine for Sepofarsen in Leber Congenital Amaurosis 10 LEIDEN, … after a single treatment with antisense oligonucleotide sepofarsen: a case report ” describing durable vision …
Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well tolerated
Agreement provides ProQR with initial payment of €12.5M and up to €135M in further payments, as well as potential additional earn outs based on commercial sales in the US and EU
Divestment of sepofarsen and ultevursen supports ProQR’s strategic focus on the Axiomer®&
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.
… ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Extension Trial Data at the Association for … from the Company’s Phase 1/2 InSight extension trial of sepofarsen and live Q&A session at the Association for …
Call on July 20 will include a snapshot from the ongoing InSight open-label extension study of sepofarsen, including preliminary second eye treatment data
