LIPID NANOPARTICLES

Acuitas specializes in the development of lipid nanoparticle delivery systems for molecular therapeutics. Our scientists have synthesized over 500 novel cationic lipids which have been evaluated in in vivo models including non-human primate (NHP) models. This evaluation characterizes the potency and safety of these LNP carriers for delivery of nucleic acid payloads including siRNA, mRNA, plasmid and antisense oligonucleotides. We have shown that the ionizable cationic lipid component critically determines the potency of LNP therapeutics. Using a rational design approach we have synthesized over 500 novel cationic lipids and screened these in an in vivo model system (Semple et al., Nat Biotechnol. 2010). We have also undertaken biophysical characterization of such novel LNP systems and conducted correlation analyses to determine which structural and biophysical properties of the cationic lipid are critical for activity. This rational design and screening approach has resulted in the identification of LNP compositions with greatly improved potency and therapeutic index (Jayaraman et al., Angew Chem Int Ed Engl. 2012; Maier et al. Mol Ther. 2013). Acuitas LNP systems are clinically validated. In a collaboration with Alnylam Pharmaceuticals, we developed the LNP carrier for Onpattro™ which has been approved for the treatment of transthyretin amyloidosis in the United States,  Europe, Japan and elsewhere. Our discovery and development programs are ongoing with the mandate to build upon our leadership position in LNP technology.

• Lipid nanoparticles for intracellular delivery of nucleic acid therapeutics
  • Messenger RNA therapeutics
  • RNAi therapeutics (siRNA and microRNA)
  • DNA therapeutics (plasmids and DNA constructs)