Search

… continue to make good progress with our three different development programs, all RNA oligonucleotides for serious … with LCA10 due to the p.Cys998X mutation received orphan drug designation (ODD) from both the U.S. Food and Drug … We believe that James’ broad knowledge and expertise in drug development and pharma will be of significant value to …

… programs that include QR-110 for LCA patients moving into development and the advancement of our discovery programs in … to the Supervisory Board through his extensive career in drug development and pharma. From 2012 until his retirement …

… and dialogue. The program is intended to optimize development plans and expedite the review and approval … injections in the eye and has been granted orphan drug designation in the United States and the European … the risks and uncertainties inherently associated with drug development, including that any one or more of our …

… a Business Update Key updates Major progress in the QR-010 development program for CF with presentation of positive … in our NPD biomarker study, showing clinically meaningful drug activity in homozygous F508del CF patients, we look … We believe that James’ broad knowledge and expertise in drug development and pharma will be of significant value to …

… QR-421 and QR-411 for Usher syndrome received orphan drug designation (ODD) from FDA and EMA. LEIDEN, The … Therapeutics as a privately-held company focused on the development of therapies for central nervous system (CNS) … in March 2017 with extensive experience in rare disease drug development, translational medicine and RNA …

… presented at the ARVO 2017 meeting.  QR-110 has orphan drug designation (ODD) in the U.S. and Europe. Partnership … $7.5 million in funding for the pre-clinical and clinical development of QR-421a for Usher syndrome type 2A targeting … Dr. Rodman has extensive experience in rare disease drug development, translational medicine and RNA …