Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025
Axiomer activity demonstrated across multiple preclinical in vitro
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 …
… 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 …
LEIDEN, the Netherlands, May 23, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced the appointment of Dr.
