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Oral delivery of genes that encode therapeutic proteins

  June, 28 2001 17:16
your information resource in human molecular genetics
Press release by Genteric Inc.:

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."

Challenges with Conventional Gene Delivery

Conventional delivery of therapeutic genes to patients involves an injection directly into the bloodstream or tissue. This direct injection method has several barriers, including: 1) difficult and inefficient gene delivery to target cells; 2) unpredictable and quickly diminishing gene activity; and 3) adverse reactions from the patient's immune response to the vector, or "vehicle," used to deliver the genes.

Genteric's Gene Pill Approach

Researchers from Genteric have invented a new method of gene delivery that employs the patient's own gastrointestinal tract to produce and secrete therapeutic proteins into the bloodstream. In the therapy's mature stage, the patient could swallow a pill containing engineered DNA that encodes the needed protein. The DNA would then be "taken up" by the cells lining the luminal space of gastrointestinal tract (the side of the intestine that normally absorbs food material), which then produce and secrete proteins into the bloodstream to achieve the desired therapeutic results. A key feature of these intestinal cells is that, like skin cells, they are constantly replaced. Thus, the gene is expelled with the dying cells on a regular basis - about every other day. This natural feature enhances safety and efficient dose control.

This new oral gene therapy method presents the following advantages:

Oral administration is simple, painless and convenient.
Oral administration allows different dose levels to be given according to patient needs.
The body replaces cells at this site quickly, which gives greater control of gene delivery and helps to avoid long-term side effects.
Genteric's next steps are to continue current experiments and follow into Phase I/II clinical trials. During these stages, researchers will address challenges in oral delivery, such as methods to adjust the amount of insulin expressed and durability of coating materials used to protect the DNA as it is delivered to cells in the intestinal tract.

About Genteric, Inc.

Established in 1997 , Genteric is a privately held biotechnology company, which owns exclusive rights to several proprietary technology platforms invented at both the San Francisco and Davis campuses of the University of California. The company has pioneered new methods of gene-based drug delivery that employ the secretory organs of the patient's gastrointestinal system to produce and release therapeutic proteins into the bloodstream.

Three of the scientific Founders of Genteric, Stephen S. Rothman, professor of physiology at UCSF; Michael S. German, M.D., associate professor of medicine in UCSF Hormone Research Institute; and Ira Goldfine, M.D. UCSF professor of medicine, invented the method that employs the patient's own gastrointestinal tract to produce and secrete therapeutic proteins into the bloodstream. Along with Michael H. Nantz, Ph.D., professor of chemistry at UC Davis, they founded the company three years ago. Dr. Rothman presently serves as Chair of the Company's Scientific Advisory Board. Both he and Dr. Nantz serve on the Board of Directors.

The company encourages inquiries regarding licensing and partnering opportunities for its technologies.

Homepage: http://www.genteric.com/

Message posted by: Frank S. Zollmann

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