Engineering a versatile solution to a long-standing problem

Pipeline

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.

Powerful parts for an even greater whole

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.

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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.

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The binding of fatty acids to albumin prevents rapid clearance by the kidney, enabling the drug to stay in the bloodstream longer and get exposure to target tissues throughout the body.

Cell

All cells have fatty acid receptors, and by linking multiple fatty acids to siRNA our technology is capable of driving efficient cellular uptake across a wide range of cell types and tissues to address previously untreatable diseases.

Syringe

Their small size relative to the siRNA keeps doses low relative to other delivery mechanisms such as antibodies, enabling both IV and convenient subcutaneous administration and keeping manufacturing costs very reasonable.

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Building better RNA therapeutics from the ground up

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.

FATTY ACIDSSpecific type of fatty acid and number of fatty acids are optimized based on the ability to enhance biodistribution and to be taken up by cells efficiently.
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LINKERConnects the siRNA to the fatty acid without interfering with siRNA uptake or performance.
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siRNAOptimized to potently and specifically silence the disease-causing gene.
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Watch how FALCON can treat disease

By pairing naturally occurring fatty acids with potent siRNAs, FALCON can target and destroy disease-causing RNA in tissues across the body.