Thursday, July 3, 2008

Lemelson-MIT Prize winner DeSimone courts Hub RNAi firms

By Ryan McBride

Joseph DeSimone came to Boston from his home state of North Carolina last week to accept the $500,000 Lemelson-MIT Prize, one of the most coveted accolades for scientific achievement. Yet DeSimone has been a more frequent visitor to the Hub for a year in search of a different prize: a deal with one of the area’s RNA-interference (RNAi) companies.

DeSimone, a chemistry professor at the University of North Carolina, Chapel Hill, is the founder and chief scientific officer of Liquidia Technologies Inc., a Research Triangle Park, N.C., startup focused on the development of nanoparticles for life sciences and industrial applications. He said the particles could be used in RNAi drug delivery — a major hurdle to making the gene-silencing treatments into lucrative products.  

Massachusetts has gained international attention for RNAi therapeutics due to University of Massachusetts Medical School professor Craig Mello’s 2006 Nobel Prize for his co-discovery of the science, and Cambridge-based Alnylam Pharmaceutical Inc.’s (Nasdaq: ALNY) leadership in the field. In lab experiments, RNAi molecules silence all types of genes linked to diseases.

DeSimone said he has visited with RNAi startups in New England such as Worcester-based RXi Pharmaceuticals Inc. (Nasdaq: RXII), of which Mello is co-founder. No deals have been disclosed. Yet DeSimone and Liquidia’s focus on the problem of RNAi drug delivery has drawn attention from fellow experts.

“My sense is he’s a smart guy and they’ve got a lot of expertise in nanoparticulate delivery systems,” said Daniel Anderson, an MIT researcher at the Koch Institute for Integrative Cancer Research. It’s unclear exactly how DeSimone and Liquidia’s nanoparticles would solve this problem. Though he did say the particles — to which RNAi molecules would be attached — are able to be shaped to enter specific types of cells.