The Cystic Fibrosis Foundation has awarded Genzyme Corp. (Cambridge, MA) a $1.05 million grant for research to identify potential small-molecule drugs to treat cystic fibrosis. The grant is the largest ever made under the foundation's Therapeutics Development Grants Program.
Cystic fibrosis is the most common fatal hereditary disease among Caucasians in the US, affecting an estimated 30,000 children and young adults. It is caused by a defect in a protein that transports chloride ions across the membrane of epithelial cells lining certain organs in the body. The most debilitating result of the defective protein occurs in the lungs of cystic fibrosis patients, where insufficient chloride transport prevents water from exiting epithelial cells. This causes the lungs to produce abnormally thick, sticky mucus that clogs the airways and is associated with fatal lung infections.
Genzyme's research will include the development of high-throughput screens to identify compounds with the potential to improve chloride ion transport or repair the particular protein defect that causes cystic fibrosis in most patients. In addition, Genzyme will employ one of its existing high-throughput screens to look for compounds that may act as agents against the most common type of bacteria found in the airways of patients with cystic fibrosis. If a product results from Genzyme's research, the company will reimburse the Cystic Fibrosis Foundation the full amount of the grant and will pay limited royalties on product sales.
To date, Genzyme has focused on two approaches to correct the basic defect in cystic fibrosis cells: gene therapy and protein replacement therapy. Genzyme has completed nine gene therapy clinical studies.
Genzyme's research to identify potential small-molecule drugs will complement these approaches. The company's drug discovery program includes combinatorial chemistry technology, high-throughput screens, and a library of over 1.5 million compounds. Small-molecule drugs may offer potential manufacturing and delivery advantages over other therapies.
Genzyme Corp. is a biotechnology and health care products company.
For more information: Henri A. Termeer, CEO, Genzyme Corp., One Kendall Sq., Cambridge, MA 02139-1562. Tel: 617-252-7500. Fax: 617-252-7600.