… of ProQR. "We have advanced QR-010 through preclinical development and are looking forward to starting the clinical … its Therapeutic Development Network, a network of CF key opinion leaders, as well as to an extensive clinical trial …
… in mice in vivo via modulation of ANGPLT3 protein properties leading to favorable increase in LPL enzymatic … lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … the potential of Axiomer EONs in preparation for clinical development and supporting discovery of potential new …
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 …
… 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 …
… lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … Davis, as Chief ADAR Scientist. Dr. Beal, one of the top experts in ADAR and RNA biology and chemistry, has been a … on his existing contributions to ProQR by driving the development and optimization of the Company’s ADAR editing …
… 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 … additional targets, along with an option for Lilly to opt in for more,” said Daniel A. de Boer, founder and CEO of …
