… 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, …
… lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today … models. “The progress the field and ProQR are making in optimizing ADAR for therapeutic use is exciting,” said Peter … income from partnerships, including the potential Lilly opt-in fee of $50 M for 5 additional targets, milestone …
… 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 …
… 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 …
