By Kim Coghill
Structural GenomiX (SGX) and the Cystic Fibrosis Foundation entered a five-year collaboration worth up to $13 million to decipher the 3-dimensional structure of a protein that, when defective, can cause cystic fibrosis.
The agreement calls for SGX, of San Diego, to unravel the structure of the transmembrane protein, CFTR (cystic fibrosis transmembrane regulator), in order to provide new insight into the disease that could lead to development of novel compounds.
"It would be very helpful if one could find out what the protein looks like in a 3-dimensional space because then you would be able to understand a little more about what causes it to malfunction," said Tim Harris, president and CEO of SGX.
People with cystic fibrosis have a defective gene that codes for the CFTR protein. That causes them to develop a thick, sticky mucus that leads to a cascade of damage to the airways and digestive tract, and a substantially increased risk of serious infections. Researchers hope to develop therapies by correcting the defective CFTR protein.
The foundation has committed $11 million to the collaboration, with additional payments for milestones and royalties.
SGX will use its genomics approach to exploit sequence variations in the CFTR gene across species to lead to an X-ray structure of the protein. CFTR plays a role in transporting chloride and regulating sodium movement within cells. No 3-dimensional structure of CFTR currently exists.
"The Cystic Fibrosis Foundation is very creative and takes a lot of initiative in forming relationships with small biotechnology companies to carry out some of its research activities," Harris said. "This was a logical extension of some of the things they've done already and we believe it to be a vote of confidence in our ability to do structural biology and structural studies on pretty complicated proteins."
SGX was founded in April 1999 by Harris, former senior vice president of research and development of Axys Pharmaceuticals Inc., of South San Francisco, and Barry Honig and Wayne Hendrickson, both professors of biochemistry and molecular biophysics at Columbia University in New York. The fourth founder is Linda Grais, a physician and attorney who has represented several biotechnology companies.
The company integrates advances in genomics, bioinformatics and X-ray crystallography into a high-throughput structure determination platform that transforms DNA sequence into 3-dimensional protein structures.
SGX, a private company, has raised $85 million in the past 14 months. n