Short interfering RNAs (siRNAs) are a powerful therapeutic modality that inhibit the expression of disease-causing genes in the nucleus and cytoplasm, offering several advantages over other types of RNA therapeutics. However, their advantages have not been fully leveraged to date because they are limited by poor biodistribution and cellular uptake. siRNAs are cleared too quickly by the kidney to be efficacious, meaning they never have the chance to reach tissues like peripheral nerves, muscle and heart, and they are not efficiently taken up by cells without a chaperone.
Our versatile FALCON (Fatty Acid Ligand Conjugated OligoNucleotides) platform conjugates combinations of naturally occurring fatty acids to siRNAs to improve their cellular uptake and biodistribution. This enables FALCON siRNAs to silence disease-causing genes in tissues beyond the liver and address disease throughout the body.