The biggest hurdle limiting the expansion of oligonucleotides as a therapeutic class is delivery–finding safe and effective ways to get siRNA into cells outside of the liver. Overcoming this challenge would enable access to undruggable targets implicated in autoimmune disease, neurodegenerative disease, cancer, glaucoma or metabolic disease. In addition to accessing validated, but undruggable targets, there are specific advantages to the siRNA mechanism with respect to duration of action and efficacy that differentiate them from small molecules, antibodies and even other oligonucleotide mechanisms.
DTx has developed proprietary technology based on lipidation, the direct conjugation of long chain fatty acids to siRNA, that enables delivery of siRNA therapeutics across multiple cell types. This technology enables oligonucleotide therapeutics to compete with small molecules and other biologics across most indications and additionally, enables access to drug targets and indications that small molecules and biologics could never be able to modulate. For additional information, contact us.