ProQR Therapeutics, RSRT join forces with Axiomer RNA editing collaboration

ProQR Therapeutics NV announced a collaboration with the Rett Syndrome Research Trust, RSRT, that will focus on the design and development of editing oligonucleotides, EONs, using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2, MECP2, and correcting mutations of interest. “The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Axiomer has the potential to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome. This collaboration marks an important step in accelerating the development of Axiomer in CNS, one of our initial areas of strategic focus along with liver-originated diseases.” RSRT awarded ProQR approximately $1 million as a research grant for the initial phase of the project, which will encompass editing oligonucleotide design and optimization, including evaluation in in vivo models for editing efficacy and MECP2 protein recovery.