Lysine-based cell-penetrating peptides for intracellular delivery
Cell-penetrating peptides for intracellular delivery.
Using lysine instead of arginine in cell-penetrating peptides (CPP’s) creates a delivery vehicle with different properties to the CPP – ASO conjugates in clinical trials. High levels of correction can be achieved with improved delivery and reduced toxicity.
Applications: Pharmaceuticals, biopharmaceuticals, drug delivery, RNA therapeutics, bioimaging, diagnostics, gene therapy
| Features | Benefits |
|---|---|
| Synthetic CPP suitable for delivery of a range of small and large molecules | Can deliver nucleotides, peptides, small molecules, or antibodies intracellularly |
| Exon-skipping correction leads to wild-type levels of expression in disease models | Effective disease modification in vivo |
| The efficacy of this class of peptides is driven purely by lysine, with no arginine in the peptide | This results in different characteristics compared to CPP’s in the clinic, and can therefore provide an alternative efficacy and toxicity profile |
| Effective biodistribution | High levels of delivery to tissues throughout the body, including improved muscle, heart and brain delivery |
| Different toxicity profile to peptides in the clinic | Overcomes issues seen with other CPP’s. The data shows that the nephrotoxicity associated with arginine-based CPP’s is unlikely |
| Wider therapeutic window compared to arginine-CPP’s | Reduced dosage required and therefore costs |
Patented and Available For
- Co-development
- Consulting
- Licensing