
Thursday, March 5, 2009
Alnylam. MIT show data on new nanoparticles
By Marc Songini
Alnylam Pharmaceuticals Inc., an RNA interference (RNAi) drug maker, is highlighting new research around a particular class of organic molecules that can be formed into nanoparticles.
Lipidoids are a new class of lipid-based molecules that can be formed into new nanoparticle compounds for systemic delivery of RNAi treatments. Cambridge-based Alnylam (Nasdaq: ALNY) announced it had published new data in a scientific journal, with collaborators from the David H. Koch Institute for Integrative Cancer Research at MIT. The new research shows indications of how to craft delivery mechanisms more precisely for small interfering RNAs (siRNAs), the molecules that mediate RNAi.
Alnylam’s and its partners’ research increased the understanding how lipidoids function and may help Alynlam as it seeks to maximize RNAi’s in vivo effectiveness. The researchers learned how to understand the long-term stability of a formulation and how to identify key parameters affecting the pharmacodynamics involved.
Researchers also showed that lipidoid formulations achieve delivery of more than 90 percent of the administered siRNA dose to the liver, while maintaining in vivo activity after several months of repeated administration.
Last January, Alnylam won U.S. Food and Drug Administration clearance to begin enrolling patients for its Phase 1 study of a liver cancer treatment.




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