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| MIT's nanoparticles (red) delivering RNA (green) to cells (blue)--Courtesy of MIT |
Alnylam has been at the forefront of the RNAi drug resurgence in the Big Biotech arena, locking down a crucial deal with Sanofi ($SNY) early in the year and several more prior.
And the company is heavily invested in the early research end, as well: scientists from Alnylam ($ALNY) teamed with MIT researchers to develop a new nanoparticle platform with which to better deliver the genetic material to the liver.
With MIT professor Robert Langer as one of its authors, the team published a paper in the Proceedings of the National Academy of Sciences highlighting its particles, which are inspired by the vehicles the body uses to transport cholesterol. The MIT-Alnylam team's particles are designed to hold the small strands of RNA and target the liver, knocking down the genes there that are linked to disease.
The nanoparticles are made of a shell of many molecules with fatty tails that face inward and proteins that face outward, according to MIT. This structure, common among nanoparticles and natural vehicles alike, holds the small RNA strands on its surface.
"What we're excited about is how it only takes a very small amount of RNA to cause a gene knockdown in the whole liver," said lead author Daniel Anderson of MIT. "The effect is specific to the liver--we get no effect in other tissues where you don't want it."
In a mouse study for the knockdown of a gene that codes for the production of a blood clotting protein called Factor VII in the liver, one of the team's compounds silenced the genes 5 times more efficiently than any other RNA vehicle.
And in a test on tumor suppressor genes, the RNA delivery was very specific to the liver, which is important in preventing side effects. The liver, though, is more conducive to this type of delivery, and the team is now looking at a way to target other organs in the body, which is a more difficult prospect.
- here's the MIT announcement