… lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … These presentations will highlight the Company’s proprietary Axiomer RNA editing platform and pipeline … ADAR RNA Editing Platform - Editing oligonucleotides optimization for therapeutic use Presenter: Peter A. Beal, …
… for AX-0810 AX-0810 is an editing oligonucleotide (EON) development program targeting the SLC10A1 RNA using the … biomarker bile acids 72 hours after treatment. Further optimizations for EONs targeting NTCP have enabled … models and showed translatability across models. ProQR is developing its AX-0810 program targeting NTCP for Cholestatic …
… le Professeur Samuel G. Jacobson, M.D., Ph.D., qui est l’ophtalmologiste qui s’occupe de quatre des patients inclus … » devrait être lancée au cours du premier semestre 2019. À propos de l’amaurose congénitale de Leber 10 L’amaurose … occidental souffrent de LCA10 à cause de cette mutation. À propos du QR-110 Le QR-110 est un premier oligonucléotide …
… Study of Homozygous F508del Cystic Fibrosis Patients Topline data to be presented today at NACFC LEIDEN, the … 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 and fast-track status by the FDA. The QR-010 …
… with no serious adverse events QR-421a is the second ophthalmology program where clinical activity was predicted … benefit was observed across multiple relevant measures appropriate to the severity of the patient’s disease, … treated eye, <5 dB.steradian change in untreated eye], and optical coherence tomography (OCT) assessment of …
… concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a … endpoints, the dose, dosing interval, and patient population for the Phase 2/3 pivotal trials,” said Aniz … were categorized into “advanced” and “early-moderate” populations based on baseline visual acuity. In advanced …
… ProQR Announces Positive Top-Line Results from a Phase 1b Study of QR-010 in Subjects … Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, added, “QR-010 exceeded … QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow ® is a …