Optimer®-enabled delivery of therapeutic cargo to a specific hepatic cell population
Customer need: Challenges with the targeted delivery and internalisation of oligonucleotide therapeutics to specific liver cells meant our partner required Optimer® delivery vehicles to increase the effectiveness of this potential new therapeutic.
Aptamer Group approach: Working with our partner we developed Optimer® binders that are highly selective to the required population of liver cells. This was performed using a hypothesis-free approach, without a priori knowledge of specific biomarkers. Optimer® discovery was driven by phenotypic differences between the target liver cell population and negative control cell populations to identify Optimer delivery vehicles that could support targeted gene therapy approaches.
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Customer benefit: The developed Optimer delivery vehicle was highly selective for the required primary liver cell population. Simple conjugation enables controlled Optimer-therapeutic cargo ratios, while the Optimer-conjugate improved targeted delivery and internalisation of the therapeutic oligonucleotide.