Kerry Benenato

TL;DR: Screening a panel of proprietary biodegradable ionizable lipids for both expression and immunogenicity in a rodent model shows that mRNA vaccine tolerability can be improved without affecting potency.

TL;DR: Clear structure-activity relationships were developed that resulted in a new amino lipid that affords efficient mRNA delivery in rodent and primate models with optimal pharmacokinetics and is the first example of the ability to safely repeat dose mRNA-containing lipid nanoparticles in non-human primate at therapeutically relevant levels.

TL;DR: Systemic administration of human PBGD mRNA encapsulated in lipid nanoparticles ameliorates disease phenotypes in mouse and rabbit models of acute hepatic porphyria and is safe in nonhuman primates, providing proof-of-concept for systemic hPBGD mRNA as a potential therapy for AIP.

TL;DR: These studies across species demonstrate preclinical proof-of-concept of systemic mRNA therapy for the treatment of Fabry disease and this approach may be useful for other lysosomal storage disorders.

TL;DR: The disclosure features novel lipids and compositions involving the same, as well as additional lipids such as phospholipids, structural lipids, and PEG lipids as discussed by the authors.