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Lipid nanoparticles and mRNA used to treat hereditary blindness

Drug Discovery World

They will lead research into using LNPs to deliver a gene editing tool that could delete bad genes in the photoreceptor cells and replace them with correctly functioning genes. . AAV has limited packaging capacity compared to LNPs and it can prompt an immune system response,” Sahay said.

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Start-up aims to advance transformative genetic medicines

Drug Discovery World

The company’s protein nanoparticle (PNP) genetic medicine delivery platform is based on the discovery of endogenous, human proteins derived from retroelements that can self-assemble to form capsid-like structures and which can package and transfer nucleic acid cargo.