Search

  3. ProQR Announces Publication in Nature Medicine of QR-110 Data for Leber’s Congenital Amaurosis 10

    Press release

    … PQ-110-001 Phase 1/2 Trial PQ-110-001 is a first-in-human open-label trial that enrolled 5 children (age 6 - 17 years) … and 6 adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients … of visual function and retinal structure through ophthalmic endpoints, such as visual acuity (BCVA), mobility …

  5. ProQR Announces Preclinical Proof of Concept Data for AX-0810 Axiomer™ RNA Editing Program Targeting NTCP for Cholestatic Diseases

    Press release

    … 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 …

  6. ProQR annonce la publication dans Nature Medicine des données du QR-110 pour l’amaurose congénitale de Leber 10

    Press release

    … 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 …

  7. ProQR Announces that QR-010 Meets the Primary Endpoint in a Proof of Concept Study of Homozygous F508del Cystic Fibrosis Patients

    Press release

    … 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 …