Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025
Axiomer activity demonstrated across multiple preclinical in vitro
… milestones achieved in H1 related to progress in the Lilly partnership €96.2 million cash position as of end of Q2 … first half of the year. With the benefits of a productive partnership, a differentiated pipeline strategy rooted in …
… of Axiomer™ for CNS applications via new research partnership with Rett Syndrome Research Trust Completed … 2024 along with potential milestone income from existing partnership, and potential option to exercise for expansion …
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