(CAMBRIDGE, Mass. and BETHESDA, Md.) -- Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the drug discovery and development affiliate of the Cystic Fibrosis Foundation (CFF), and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that they have initiated a collaborative program to discover Direct RNAi(TM) therapeutics for the treatment of cystic fibrosis (CF).
Alnylam will apply RNAi technology toward the discovery of short interfering RNAs (siRNAs), the molecules that induce
Cystic fibrosis is a
"Existing drugs for cystic fibrosis focus on the symptoms and complications of the disease, such as preventing infections, reducing the quantity and thickness of secretions in the lungs, and improving breathing and
This genetic disease is marked by defects in a protein, known as the Cystic Fibrosis Transmembrane conductance Regulator (
In most patients, potentially functional
"The opportunity to harness RNAi to treat CF represents an important strategy in the application of our technology for discovering novel medicines and making a difference in patients' lives," said John Maraganore, Ph.D., president and CEO of Alnylam Pharmaceuticals. "The ability to collaborate with a strong foundation, such as the CF Foundation and its affiliate, CFFT, allows us to address this unmet medical need. We believe applying our technology to this disease represents an outstanding opportunity for Alnylam and look forward to bringing the potential fruits of this collaboration to patients."
About the Cystic Fibrosis Foundation and CFFT
The mission of the Cystic Fibrosis Foundation is to assure the development of the means to cure and control CF and to improve the
About RNA Interference (RNAi)
RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence and regulate such genes selectively through RNAi could provide a means to treat a wide range of human diseases. The discovery of RNAi has been heralded by many as a major breakthrough, and the journal Science named RNAi the top scientific achievement of 2002, as well as one of the top 10 scientific advances of 2003.
About Alnylam Pharmaceuticals, Inc.
Alnylam is a biopharmaceutical company seeking to develop and commercialize novel therapeutics based on RNA interference, or RNAi. Growing from its foundation as the world's first company focused on RNAi therapeutics, the company's leadership in the field of RNAi is supported by its preeminent founders and advisors and its strengths in fundamental patents, technology, and know-how that underlie the commercialization of RNAi therapeutics. Alnylam is developing a pipeline of RNAi products using Direct RNAi(TM) to treat ocular, central nervous system, and respiratory diseases and