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
… 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
Access based on positive interim analysis of clinical data as well as preclinical data to date PRIME designation provides a pathway for frequent and early interactions with the EMA aimed at supporting accelerated evaluation and approval
… 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 …
