… in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, … on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and … Principal Researcher at ProQR "I work on designing and optimizing new therapies" Nanyamka Braham from Regulatory …
… 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 …
… As an employer, we value a diverse workforce in which people are encouraged to develop themselves both personally and professionally. We … 30 vacation days An annual performance bonus Stock options A premium-free pension plan A commuting allowance A …
Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseasesAdvancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett SyndromeHosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 P
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened leadership with appointments of Chief Financial Officer and Chief
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multi
… 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 …
Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight dataMartin Maier, PhD nominated to Board with Annual General
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
AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024$4.5 million in milestones
