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Optimer®: Accelerating your journey to clinic

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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.

Fluorescence microscopy analysis demonstrates selective targeting of the Optimer® delivery vehicles to the intended primary liver cell population with no targeting to alternative primary hepatic cell populations.

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.