… ProQR Announces Additional Sepofarsen Illuminate Trial Analyses and Provides Update on Company Strategy … further potential. ProQR will present further non-clinical data for Axiomer ® and announce its internal … there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated …
Divestment of sepofarsen and ultevursen ophthalmic programs supports ProQR’s strategic focus on Axiomer® RNA editing platform technology and continued advancement of pipeline
€129 M cash and cash equivalents as of June 30, 2023 providing runway into mid-2026
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas
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… AX-0810 targeting NTCP and AX-1412 targeting B4GALT1 into clinical development in late 2024/early 2025. Financial … intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner … continued investment in it, as well as the timing of our clinical development, the potential of our technologies and …
ProQR is accelerating the development of its Axiomer® RNA base-editing technology platform across multiple therapeutic areas and will provide an update on first targets in H2 2022
Company plans to discuss findings from sepofarsen Illuminate trial w
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators and pr
Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in vision; Phase 2/3 pivotal trial Illuminate initiated with data expected in H1 2021Phase 1/2 St