Arcturus Therapeutics

Technologies

We believe our two proprietary technology platforms solve major hurdles in RNA medicine development such as
delivery challenges, limited potency, and narrow therapeutic index.

LUNAR®

Our first platform is a novel lipid-mediated delivery system called Lipid-enabled and Unlocked
Nucleomonomer Agent modified RNA (LUNAR®). LUNAR is a multi-component drug delivery system that is versatile and
with our library of over 150 proprietary lipids, diverse. Our preclinical studies have shown that formulations can
be customized for the indication and target cell type of interest. We have also demonstrated that our formulation
process is scalable and reproducible.

 

LUNAR®-mediated delivery of RNA into cells

LUNAR particles associate with the cell membrane of a target cell of interest and quickly enter the cell via endocytosis.

Upon entering the cytosol, the LUNAR formulations then become trapped in the endosomes.

With increased acidity as the endosome ages, a pH-mediated disruption enables release of the RNA payload following rapid biodegradation of the LUNAR components.

Once release of the RNA into the cytosol occurs, the translational machinery can interact with the RNA and processing and/or trafficking can then take place to make functional protein.


Benefits of LUNAR®

  • LUNAR delivery technology is not limited to a specific RNA modality. Preclinical studies have
    demonstrated compatibility with different types of RNA, from short double-stranded siRNAs, to long
    single-stranded mRNAs, to double-stranded DNA molecules. This means that we are not restricted in the types of
    RNA medicines that we can develop.
  • LUNAR® delivery technology is compatible with multiple routes of administration and can be targeted to diverse
    tissues and cell types. Preclinical studies in both rodents and non-human primates have shown that LUNAR can
    deliver RNA compounds specifically to hepatocytes and stellate cells via intravenous injection,
    muscle cells via intramuscular injection, and lung cells via nebulization.
  • Ability to repeat dose. Multiple preclinical studies in rodents and non-human primates have shown no reduction
    in efficacy upon repeat dosing of LUNAR formulated siRNA or mRNA.

 

UNA

Our second platform is a novel oligonucleotide chemistry technology called Unlocked Nucleomonomer Agent (UNA). UNA can
be used to reduce off-target effects and increase duration of action in siRNA medicines.

UNA

 

  • Arcturus’ UNA oligomer chemistry can improve the in vivo efficiency of small interfering RNA (siRNA)-mediated
    gene silencing and potentially improve the efficiency of messenger RNA (mRNA)-mediated protein replacement.
  • One or more UNAs can be positioned strategically along a RNA strand to manipulate the chemical
    properties of the molecule. Addition of a single UNA in siRNA therapeutics can enable more efficient suppression
    of target protein production and reduce off-targets effects.

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