Alnylam nabs CF funding

BETHESDA, Md.—After a few years of funding academic research and observing advances in the burgeoning field of RNAi, Cystic Fibrosis Foundation Therapeutics (CFFT) Inc., recently announced its first funding of a commercial partner to initiate discovery research for a potential RNAi therapeutic for CF. Alnylam Pharmaceuticals Inc., of Cambridge, Mass., will receive $1.5 million in up front and milestone payments over the next year to identify specific short interfering RNAs (siRNAs) as potential therapies for cystic fibrosis.

"We decided to work with Alnylam because they have the appropriate licenses to use siRNA as a therapeutic and they are well-positioned from the IP perspective to take the next step and look at siRNA as an actual therapeutic," says Diana Wetmore, CFFT vice president of alliance management.

Of particular note, Wetmore says, was the IP related to the chemistry Alnylam performs for chemical modification of nucleic acid to improve its stability. "I felt that this method was one that would make a difference for the chance of success of their molecule," Wetmore says.

Barry Greene, COO of Alnylam, counts CFFT among a number of similar research partnerships, including a 2003 agreement with Merck to further the development of RNAi technology and a second Merck collaboration announced last year aimed at developing potential RNAi therapies for

age-related macular degeneration (AMD).

"We are very excited to have an alliance with the Cystic Fibrosis Foundation," says Greene. "Cystic fibrosis is a horrific disease and no therapies exist that target cystic fibrosis itself. We believe, consistent with learning we have made out of our own R&D activities aimed at the lung, that cystic fibrosis can be a direct RNAi application."

A genetic disease, cystic fibrosis is marked by defects in a protein called the cystic fibrosis transmembrane conductance regulator (CFTR), which is required for the proper transport of salt and water across a cell membrane. This defect leads to thick sticky mucus that clogs the lungs and digestive system. Most CF patients produce potentially functional CFTR proteins and the aim of the Alnylam research is to determine whether the protein can be redirected to the cell surface by use of RNAi to silence specific genes.

While no time frame has been specified, both CFFT and Alnylam anticipate having a collection of molecules in development within 12 months. "As the program achieves success, we anticipate [CFFT] will continue to fund the program into clinical studies," says Greene. "We haven't given any guidance for this program, but within 18 months we believe we can go from a molecule of interest to one that is ready for animal testing. We can move much more rapidly with RNAi than with other approaches."