The first patent for a method of delivering normal genes in a pill to induce the production of insulin in people with diabetes was issued May 1 to the University of California, San Francisco by the United States Patent and Trademark Office.
Sometimes referred to as a "gene pill," the oral delivery of normal genes has been a long-sought and elusive technique. Now, UCSF researchers have successfully demonstrated that raw DNA taken orally can find its way inside cells lining the intestinal tract and prompt those cells to express a protein, such as insulin, even though they are not specialized for that purpose.
"Considerable work still needs to be done before there is an actual gene pill for insulin production, but the patent demonstrates the validity of the concept and promises to spur development of oral delivery of genes to treat a vast number of illnesses," said Stephen S. Rothman, PhD, professor of physiology in the UCSF Schools of Medicine and Dentistry.
The technique holds the potential for providing patients with more than 50 proteins normally secreted by the body into blood and which patients now receive by injection into muscles - including insulin, growth hormones, blood factors for treating hemophilia, and erythropoietin for treating anemia. Rothman expressed confidence that future patent awards to UCSF would cover development of the method for many illnesses.
Genteric, a biotechnology company in Alameda, Calif. has an exclusive, worldwide license agreement with the University to use the method for drug development. The inventors of the technique - Rothman; Michael S. German, MD, associate professor of medicine in the UCSF Hormone Research Institute; and Ira Goldfine, MD, UCSF professor of medicine - along with Michael H. Nantz, PhD, professor of chemistry at UC Davis, are the founders of Genteric.
The research team began studying the technique at UCSF five years ago with funding from the UCSF Foundation and
Contact: Bill Gordon
University of California - San Francisco