Gene-silencing siRNA technology has failed to live up to its original hype largely because of problems delivering short interfering RNA to the precise spot where it's needed. Alnylam Pharmaceuticals, a Cambridge, MA company focused on RNAi-based therapeutics, together with partners at MIT, think they've found a solution. A release says they have published new data describing a new approach for systemic delivery of RNAi therapeutics using combinations of lipid-like materials called "lipidoids."
The new research results, which were published in the journal Molecular Therapy, says that two lipidoids together can be combined for a kind of one-two punch "to achieve synergistic gene silencing effects," according to the release. The company said that the gene-silencing effect seemed to work both in vivo and in vitro. Also, the release said, this binary formulation strategy could be applied to any siRNA delivery material in any target cell population that utilizes combinations of unique components to mediate distinct steps in the delivery process, for example lipids, polymers and siRNA conjugates.
"These new data reflect our continued and broad-based commitment to developing novel approaches for the systemic delivery of RNAi therapeutics. In working with world-renowned academic leaders in the field of drug delivery at MIT, we continue to expand our understanding and knowledge about innovative delivery approaches," said Kevin Fitzgerald, senior director of research at Alnylam. "These recently published data show that binary combinations of specific lipidoids can result in optimized lipid nanoparticle formulations with synergistic siRNA delivery properties."
Alnylam recently filed an application to initiate Phase I trials for ALN-PCS, its second-generation lipid-nanoparticle delivery technology to treat severe hypercholesterolemia, or high cholesterol in the blood. It should also be mentioned that in March of this year, Tekmira Pharmaceuticals filed litigation against Alnylam concerning its lipid nanoparticle delivery technology.
- take a look at Alnylam's release
- and an abstract of the Molecular Therapy paper