Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohort
… 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 …
… 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 …
… NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR), a biopharmaceutical company dedicated to changing lives through … In addition, ProQR has granted the underwriters a 30-day option to purchase up to 1,363,636 additional ordinary shares … dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing …