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Alnylam, MIT Team Publishes Data on Effects of 'Lipidoid' Combos

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Alnylam Pharmaceuticals has published data on the use of combinations of novel lipid materials to systemically deliver RNAi therapeutics.

The work was conducted by Alnylam researchers in collaboration with scientists at the Massachusetts Institute of Technology (GSN 5/10/2007), and appeared in Molecular Therapy.

Alnylam said the research showed that binary combinations of so-called lipidoids can be “formulated together in a single lipid nanoparticle formulation to achieve synergistic gene silencing effects both in vitro and in vivo.

The company said that more than 3,500 novel lipid nanoparticle formulations were tested, and results showed “synergistic gene silencing when utilizing materials that, when combined, mediated both efficient cellular uptake and productive endosomal escape.”

The data also showed that “ineffective single lipidoids could be formulated together to induce robust silencing of the luciferase mRNA in vitro and the factor VII mRNA in vivo,” in HeLa cells and in mice, respectively.

"It is anticipated that this binary formulation strategy could be applicable to any siRNA delivery material in any target cell population that utilizes the two-step endosomal delivery pathway," according to the paper.

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