Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and miceFunctional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favora
… lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … neurodevelopmental disorder, affects approximately 350,000 people worldwide, predominantly girls. With no current cures and limited treatment options, Rett Syndrome presents a significant unmet medical …
… Research Partnership and EB Medical Research Foundation to Develop QR-313 for the Treatment of Dystrophic Epidermolysis Bullosa The funding will be used for the clinical development of QR-313 Clinical trial in DEB patients to start …
… are no therapies commercially available or in clinical development for this disease. QR-110 has received fast track … granted orphan drug designation in the United States and European Union. Interim safety and efficacy trial results … that the first patient has been dosed in the Phase 1/2 open-label trial assessing the safety, tolerability, …
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
… 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 …
