Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseasesInitiating Phase 1 study of AX-0810 in healthy volunteers, with initial safety, tolerability, and PK data from Cohort 1 expected by year-end, and target engagemen
AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024$4.5 million in milestones
… biomarker study for QR-504a for Fuchs endothelial corneal dystrophy to be reported in 2022 Cash runway into mid-to-late … individuals from the Phase 1/2 Stellar trial in the Helia open-label extension study, which will include multiple dose … data from this trial in 2022. ProQR has been further optimizing the Axiomer ® Editing Oligonucleotide (EON) …
… the existing proven delivery technology to the liver, the opportunity to monitor early biomarkers to establish target … in human genetics with a significantly lower chance of developing cardiovascular disease. ProQR intends to advance … the 8 th annual Oligonucleotide and Precision Therapeutics (OPT) Congress and at the bi-annual RNA Editing Gordon …
… genetic eye disease, QR-504a for Fuchs endothelial corneal dystrophy.” Business Operations and Program Updates … protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended … with advanced FECD. Currently there are no treatment options available to stop or slow down FECD and disease …
… Fuchs Focus study of QR-504a for Fuchs Endothelial Corneal Dystrophy (FECD) is currently open for enrollment. This study … protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended … with advanced FECD. Currently there are no treatment options available to stop or slow down FECD and disease …
… diseases, today announced virtual presentations at the Ophthalmology Futures Retina Forum, European Society of Retina Specialists (Euretina) congress and the Annual Meeting of the American Academy of Optometry (AAOpt). ProQR’s presentation at Ophthalmology …
AX-0810 targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to host virtual Investor and Analyst Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting
… access to the PRIority MEdicines (PRIME) program by the European Medicines Agency (EMA) for sepofarsen. The PRIME … that are for indications that currently have no treatment options. To be eligible and accepted for PRIME, a medicine … PRIME, the EMA offers additional support to medicine developers including early interaction and dialogue. The …
… received orphan drug designation (ODD) from the FDA and European Medicines Agency (EMA). QR-411 targets the … QR-010 is designed to be self-administered via an optimized eFlow ® Nebulizer (PARI Pharma GmbH). eFlow ® is a … financial statements, the potential exercise of share options is not included in the diluted earnings per share …