DTx Pharma

DTx Pharma is using RNA-based delivery mechanisms to treat genetic conditions. Short interfering RNAs (siRNAs) can be used to inhibit the expression of disease causing genes. However, one of the major challenges with using RNA therapies is attaining delivery to target tissues beyond the liver. DTx has developed their Fatty Acid Ligand Conjugated OligoNucleotides (FALCON) platform that leverages siRNAs and fatty acid pharmacology to overcome this. FALCON involves conjugating naturally occurring fatty acids to siRNAs to improve cellular uptake and bioavailability. DTx is currently using their technology to treat peripheral nervous system, muscular and CNS disorders, with their lead candidate, DTx-1252 anticipated for clinical development.

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