In June we caught up with our Lead Talent Acquisition, Silvia Catellani, to talk about ProQR, Pride, and driving an inclusive culture for present and future ProQRians.
On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.
… 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 …
… AX-1412 B4GALT1 Cardiovascular disease 2025 update on optimization for GalNAc delivery Continue to execute on … milestone income from the existing partnership, and an option to exercise for an additional five targets for … a total of 15 targets, which would result in a $50 million opt-in payment to ProQR. Financial Highlights At June 30, …
… B4GALT1 for Cardiovascular diseases mid-2025 update on optimization for GalNAc delivery Continue to execute on … milestone income from the existing partnership, and an option to exercise for an additional five targets for … a total of 15 targets, which would result in a $50 million opt-in payment to ProQR. ProQR may selectively form new …
AX-0810 target engagement data in healthy volunteers on track for Q2 2026; biliary atresia selected as initial Phase 2 indicationPipeline expansion with advancement of AX-0811 (NTCP) and AX-0422 (IDUA) toward the clinic, supporting multiple upcoming clinical cataly
… lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today … Advisory Board. As Chief ADAR Scientist, he drives the development and optimization of the Company’s Axiomer ADAR-mediated RNA …
… pipeline program, QR-504a for Fuchs endothelial corneal dystrophy, is expected to advance to clinical testing in the … visual acuity, visual fields, and the objective measure of optical coherence tomography (OCT) retinal imaging, after a … The closing included the full exercise of underwriters’ option to purchase 2,076,923 additional shares. Gross …
… biomarker bile acids 72 hours after treatment. Further optimizations for EONs targeting NTCP have enabled … AX-0810 targeting NTCP for cholestatic diseases: clinical development candidate translational data to be reported, and … enable the suppression of nonsense mutations and premature stop codons, which can edit a uridine (U) into a …
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
