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Gene Drugs For Untreatable Diseases
Kerry A. Dolan, 05.14.08, 6:00 PM ET

BURLINGAME, CALIF. - Working from a quiet office park across from a railroad terminus in Richmond, Calif., scientists at Sangamo BioSciences have compiled a hefty pipeline of drug candidates utilizing zinc-finger DNA-binding proteins, which can manipulate genes precisely or delete them altogether.

Sangamo BioSciences (nasdaq: SGMO - news - people ) is going after conditions and diseases for which there are no good drug treatments, including amyotrophic lateral sclerosis (Lou Gehrig's disease) and spinal cord injuries.

Turning on specific genes could, for example, enable nerve regeneration or blood-vessel growth in ways that drugs thus far have been unable to. Correcting malfunctioning genes could perhaps cure diseases linked to a single gene, such as sickle-cell anemia.

So far, Sangamo has a drug in mid-stage clinical trials for two different ailments. The furthest along is a treatment for the lower-leg nerve damage caused by diabetes. Diabetic neuropathy affects 10 million people in the U.S. and is the cause of at least 70% of the 100,000 amputations performed each year.

Current treatment options are painkillers and antidepressants; no drugs are approved to reverse the nerve damage that occurs when blood glucose levels remain too high for an extended period. (The condition can be reversed if patients control their blood glucose levels.)

Data from an early stage trial of 20 people suggested the Sangamo drug, called sb-509, may not only protect nerves from further damage but possibly help in nerve regeneration as well. The drug turns on the gene that stimulates production of vascular endothelial growth factor. This growth factor has been shown to promote blood vessel growth, protect nerves and promote regrowth of nerves.

Results of a larger mid-stage clinical trial are due in the second half of 2008. The Juvenile Diabetes Research Foundation is providing Sangamo as much as $3 million to support a trial of the drug in diabetes patients.

Previous attempts by other drug companies to harness gene therapy have, at times, had disastrous results. In one trial in 1999, an 18-year-old patient died; in a separate trial in France, a child developed leukemia. In both cases, the virus used to deliver the gene was believed to have caused the adverse reaction. Sangamo's drugs thus far are not being delivered using viruses, and so might be safer.

Because Sangamo Chief Executive Edward Lanphiere obtained exclusive licenses from the handful of academics working on zinc-finger proteins, Sangamo has a near lock on patents relating to zinc-finger proteins. It has 65 U.S. patents (15 of which are licensed from researchers; the rest were generated at Sangamo) and 117 foreign patents (17 are licensed) and another 89 patents applied for in the U.S.

No other companies are competing using the same technology. "This is the first new drug-development platform in the post-genomic era," beams Lanphiere.

Time will tell if it lives up to its huge promise

http://www.forbes.com/technology/ebu..._0514gene.html