… lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today … the existing proven delivery technology to the liver, the opportunity to monitor early biomarkers to establish target … models. “The progress the field and ProQR are making in optimizing ADAR for therapeutic use is exciting,” said Peter …
… Announces Encouraging AX-0810 Phase 1 Safety and PK Data, Development Candidate Selections, and 2026 Outlook Initial … lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … observations in healthy volunteers,” said Cristina Lopez Lopez, MD, PhD, Chief Medical Officer of ProQR. “These …
… European Medicines Agency Grants PRIME Access to ProQR’s … its sepofarsen (QR-110) drug candidate, which is being developed for targeting the p.Cys998X mutation in the CEP290 … been granted access, and only 20% (one out of five) of ophthalmology applications have been granted access. “The …
… and EMA, representing the third candidate in the company’s ophthalmology pipeline and the fourth in the broader pipeline … no therapies commercially available or in clinical development for the vision loss associated with Usher … disease, and QRX-504 for Fuchs endothelial corneal dystrophy. Promising QRX-421 pre-clinical data in both …
… Collaboration New agreement supports the discovery and development of additional assets directed toward high … originally announced in September 2021, applied ProQR’s proprietary Axiomer® RNA editing platform to target disorders … biodistribution in both the liver and nervous system, opening up new potential applications to not only correct …
… two abstracts on programs for Fuchs endothelial corneal dystrophy and Stargardt’s disease at the ARVO annual meeting. … patients have been enrolled in PQ-110-001, a Phase 1/2 open-label trial assessing the safety, tolerability, … treatment to LCA 10 patients that have no other treatment options,” said Daniel A. de Boer, Chief Executive Officer of …
… About the PQ-110-001 Phase 1/2 trial PQ-110-001 was an open-label trial designed to enroll children (over six years of age) and adults who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. A total … period and eligible patients are being given the option to participate in Insight, an open-label extension …
… perception de la lumière (MVAC> 3,0 LogMAR) au départ, la population étudiée dans le cadre d' Illuminate était plus … d'acuité visuelle de l'Early Treatment of Diabetic Retinopathy Study (ETDRS ou Étude sur le traitement précoce de la rétinopathie diabétique) et le Test de vision rudimentaire de …
… board, where his expertise will help guide and inform our development of therapies for rare genetic diseases based on … ® technology." Dr. Yu is a professor of biochemistry and biophysics at the University of Rochester Medical Center. His … rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. Based …
