• New non-human primate data show highly selective in vivo delivery of mRNA to T cells with a cell-targeted lipid nanoparticle (ctLNP)

CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today presented data on its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms at the European Society of Gene and Cell Therapy (ESGCT) 31^st 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 (NHPs). In the study, conducted as part of Generation Bio’s partnership with Moderna, Inc., 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.

Figure 1: Highly specific transduction of CD3+, CD4+, and CD8+ cells with T cell targeted ctLNP achieved following 1 mg/kg dose of ctLNP-mRNA, with no monocyte uptake above baseline

“The robust and selective in vivo delivery to T cells in an NHP model gives us the conviction to pursue development of T cell targeted in vivo genetic medicines,” said Matt Stanton, Ph.D., chief science officer of Generation Bio.

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.

Dr. Stanton added: “Our research on iqDNA continues to yield important insights into the characterization and behavior of this new molecule. We are pursuing increasingly focused areas of research to make iqDNA ready for the development of novel genetic medicines.”

To learn more about the data presented at ESGCT, visit the Scientific Presentations page of Generation Bio’s website.

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