RE: Acuitas Partnership7 May 2026 09:03
The following was published by Acuitas last year, and so obviously their R&D is well advanced in this area, and perhaps no surprise they're happy to collaborate with Genflow, covering all costs and R &D expenditure, to progress from the mouse model with Genflow's Sirt6,
compelling and potential panacea payload...Gla holders...a Major re-rate beckons!!!! :-)
July 3rd, 2025
Acuitas Therapeutics Collaborates on mRNA-LNP Prime
Editing Study for Genetic Liver Disease
Vancouver, B.C. – Acuitas Therapeutics today announced its collaboration on a landmark study published in Nature Biomedical Engineering, titled “Treatment of a metabolic liver disease in mice with a transient prime editing approach,” which highlights the therapeutic potential of
lipid nanoparticle (LNP)-mediated mRNA delivery for prime editing in vivo. The study – co-authored by Acuitas scientists Ying Tam, Jennifer Moon, Paulo Lin, and Steven Fan, along with partners from the University of Zurich, ETH Zurich, and the University of Pennsylvania –presents a novel approach to correct a disease-causing mutation in a mouse model of phenylketonuria (PKU), an inherited metabolic liver disorder.
The research team used two distinct strategies for liver-targeted prime editing, both of which delivered the prime editor (PE) as nucleoside-modified mRNA encapsulated in Acuitas’ LNP.
In the first strategy, the guide RNA (pegRNA) was stably expressed from an adeno-associated virus while in the second, the pegRNA was delivered as synthetic RNAs using LNP delivery.
Using these approaches, they achieved up to 47.4% editing at the Dnmt1 locus and up to 20.7% correction of the pathogenic Pahenu2 mutation in PKU mice with the enhanced PE7 variant –successfully reducing blood phenylalanine concentrations below the therapeutic threshold of 360 µmol/L.
The transient nature of mRNA-LNP delivery allowed for high editing efficiency, while minimizing prolonged editor expression and the resultant potential off-target effects, which are concerns with viral delivery systems. These findings highlight the promise of non-viral, mRNA-LNP-based prime editing as a scalable and clinically relevant platform for treating monogenic liver diseases.