Oral therapy

GENTERIC ANNOUNCES BREAKTHROUGH GENE THERAPY IN A PILL

First Patent Ever Awarded For Oral Delivery of Non-Viral Gene Therapy
ALAMEDA, Calif. - May 3, 2001 - Oral delivery of genes that encode therapeutic proteins has long been a goal of gene therapy research. Now, for the first time this treatment to correct human disease may be possible thanks to a breakthrough gene therapy technique being pursued by Genteric, Inc. a privately held biotechnology company.

Today, the U.S. Patent and Trademark Office issued U.S. patent Number 6,225,290, for the "first ever" method of oral delivery of non-viral DNA encoding the human insulin gene. This revolutionary development called the "Gene Pill," is the exclusive property of Genteric, as a result of a licensing agreement with the University of California, San Francisco.

Researchers at the university began by introducing engineered DNA directly into the pancreas where the majority of digestive enzymes are produced. Remarkably the DNA demonstrated resilience to the enzymes in the pancreas. The team then decided to pursue DNA delivery into the gastrointestinal tract achieving the same desired effect.

Genteric's Gene Pill approach uses the patients' own gastrointestinal organs to covert genes to therapeutic proteins. The body naturally distributes the proteins without the need of a "vector system," hence achieving the desired therapeutic effect safely. Genteric's research has shown for example, that when insulin DNA is administered to the gastrointestinal tract of diabetic rats, insulin protein is produced and secreted into the bloodstream, lowering blood glucose to normal levels.

"This patent, and others which will follow, represents the ultimate therapeutic application of new protein drugs which will result from sequencing the human genome. While much work needs to be done and public availability of the Gene Pill therapy is several years away, the possibility of replacing injection-based delivery of insulin to the body with an oral gene therapy is great news to millions of diabetics who currently have no other appealing options. Genteric's innovative approach to gene delivery is among the most advanced methods being pursued today," said Martin D. Cleary, president and chief executive officer of Genteric.

According to Roland Scollay, Ph.D., Genteric's vice president of research and chief scientific officer, the Gene Pill has the potential to treat a wide array of other diseases, such as growth hormone deficiency, hemophilia, Gaucher's disease, autoimmune disease, cancer and bacterial and viral infections.

"Although we have always believed that gene-based therapies hold tremendous potential for the treatment of a variety of diseases, clinical advances have been hindered by failure to deliver therapeutic genes safely and conveniently," said Dr. Scollay. "The Gene Pill technique can provide a safe, non-systemic, non-viral vehicle for drug delivery- which has been the missing link in gene therapy. This will provide exciting new treatment options for a number of disorders."