[Alnylam: Cystic Fibrosis Foundation Therapeutics to Provide $1.5 Million]
CAMBRIDGE, Mass. and BETHESDA, Md.--(BUSINESS WIRE)--March 16, 2005--
Cystic Fibrosis Foundation Therapeutics to Provide $1.5 Million
Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the drug discovery and development affiliate of the Cystic Fibrosis Foundation (CFF), and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY - News), 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 RNA interference, to restore protein function in CF. CFFT is expected to provide Alnylam with $1.5 million in up front and milestone-driven funding for the discovery effort, along with introductions to world-class CF academic researchers and access to critical research resources.
Cystic fibrosis is a genetic disease affecting approximately 30,000 children and adults in the United States. A defective gene causes the body to produce an abnormally thick, sticky mucus that obstructs the lungs, leading to life-threatening lung infections, and obstructs the pancreas, causing difficulty absorbing food. The median life expectancy has improved from early childhood to the mid-30s today, but many individuals battle lung disease for years.
"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 digestion. We hope a potential therapeutic will result from this groundbreaking technology to treat the cause of the disease," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation and CFFT. "We are very pleased to collaborate with Alnylam, a leader in the discovery and development of RNAi therapeutics, to focus on CF."
This genetic disease is marked by defects in a protein, known as the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, required for proper transport of salt and water across the cell membrane. Defects in this protein lead to thick, sticky mucus that clogs the lungs and digestive system. In most patients, potentially functional CFTR protein is made but does not reach the cell surface. CFFT and Alnylam aim to determine whether this protein can be redirected to the cell surface by using a potential RNAi therapeutic to silence specific genes. If so, the goal will be to develop this potential RNAi therapeutic as a treatment for CF patients.
"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." <<
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Cheers, Tuck |
