… lives through transformative RNA therapies based on its proprietary Axiomer ® RNA editing technology platform, today … led to approx. 50% editing in liver of mice. New optimizations are being evaluated (not disclosed in the … prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing …
… ProQR Announces Proposed Underwritten Public Offering of Ordinary Shares … addition, ProQR expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the offering … existing cash and cash equivalents, to advance clinical development of its product candidates, to progress its other …
… oligonucleotide QR-110 for treatment of inherited retinal dystrophy in adults and children with LCA10 Presenter : David … Poster title: Structure-based computational approach for optimizing oligonucleotides for A-to-I editing Poster #: 29 … orphan drug designation in the United States and the European Union and received fast-track designation by the …
… 1b safety, tolerability and exploratory efficacy trial. Top-line data to be reported in mid-2017 Top-line data from a … of QR-010. A full study presentation is planned for the European Cystic Fibrosis Society Conference in June 2017. … QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a …
… - Nucleic Acids . LCA10 is a severe inherited retinal dystrophy associated with mutations in the CEP290 gene. … process by which QR-110 was identified as a potentially optimal therapeutic candidate using a patient-derived retinal … suggest the pharmacodynamic, pharmacokinetic and safety properties make QR-110 a promising candidate for the …
… at NACFC on October 27 Noreen Henig, M.D., Chief Development Officer of ProQR and John P. Clancy, M.D., … QR-010 is designed to be self-administered via an optimized eFlow ® Nebulizer (PARI Pharma GmbH). eFlow ® is a … orphan drug designation in the United States and the European Union. The QR-010 project has received funding from …
… Therapeutics, will present on June 20, 2024 at 2:00pm ET: “Developing Axiomer RNA Editing Technology Towards Clinical Development” This presentation will provide an overview of ProQR’s proprietary ADAR-mediated Axiomer RNA editing technology …
… Team. During the event, the Company will showcase its proprietary Axiomer ® RNA-editing technology platform, announce initial pipeline targets for internal development, and provide guidance on the advancement of … has led in the development of structure-guided methods for optimizing chemically modified oligonucleotides for …
… Board. Dr. Dryja will play a key strategic role in the development and advancement of ProQR’s growing ophthalmology pipeline. LEIDEN, The Netherlands, Nov. 09, …