12:00 AM
Jan 12, 2015
 |  BioCentury  |  Emerging Company Profile

Neutralizing RNAi

Solstice formulating ribonucleic neutrals to broaden delivery of RNA therapies

The negatively charged phosphate backbone of RNA is critical for the oligonucleotide's biological activity but also prevents its uptake into cells and limits its circulating half-life. Solstice Biologics LLC's ribonucleic neutrals platform could enable efficient systemic delivery to tissues beyond the liver, broadening the range of diseases that can be addressed with RNAi therapies.

RNAi companies have attempted to address delivery issues by using various carbohydrates and polymers as delivery vehicles to mask the charged backbone and enhance cellular uptake of therapeutic RNAs. However, these delivery vehicles balloon the mass and size of the delivered molecule, resulting in a low diffusion co-efficient that makes delivery to most tissues outside the liver inefficient and potentially unsafe.

Consequently, companies developing RNAi therapies, including leaders such as Alnylam Pharmaceuticals Inc., remain focused on targeting hepatic cells.

"We see the huge value created by Alnylam from just targeting liver indications with RNAi therapies and now we have a technology that enables us to access...

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