Generation Bio presents preclinical data on ctLNP and iqDNA

Generation Bio (GBIO) presented data on its cell-targeted lipid nanoparticle – ctLNP – and immune-quiet DNA – iqDNA – platforms at the European Society of Gene and Cell Therapy 31st Annual Congress. The company presented data showing that a single IV dose of its ctLNP delivered mRNA encoding a reporter protein to a majority of circulating T cells in non-human primates. In the study, conducted as part of Generation Bio’s partnership with Moderna (MRNA), NHPs received a single 1 mg/kg dose of ctLNP-mRNA, which transduced most T cells with balanced distribution to CD4+ and CD8+ cells. Untargeted LNPs did not elicit T cell transduction. The biodistribution of ctLNP-mRNA to the liver, spleen, lung, and monocytes was minimal. These results in NHPs confirm and improve on results from prior murine studies. The company also presented updates on iqDNA, which is a partially single-stranded DNA molecule that avoids recognition by primary DNA sensors in mice and NHPs. Recent work on the iqDNA platform has focused on methods of improving protein expression, including priming second strand synthesis by encoding the promoter region as double-stranded and stabilizing iqDNA against degradation by exonucleases.