Fatty acid motifs are a tried-and-true approach for improving biodistribution and uptake in marketed diabetes drugs like insulin detemir, degludec, liraglutide and semaglutide.
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.
We combine a deep understanding of structure-activity relationships with world-class siRNA medicinal chemistry capabilities to create highly efficacious therapies tailored to each target tissue.
Our proprietary FALCON platform optimizes the siRNA sequence, number of fatty acids, fatty acid type, and linker to safely and effectively deliver siRNA therapeutics to an unprecedented range of organs and cell types.
By pairing naturally occurring fatty acids with potent siRNAs, FALCON can target and destroy disease-causing RNA in tissues across the body.