By Allan Maurer, Tech Journal South

GAINSVILLE, FL—Transgeneron Therapeutics, winner of the Southeast BIO investor forum early-stage shootout competition in early November, is developing a “master switch” protein that reverses diabetes in mice and may do the same in humans.

Leslie Molony, Ph.D., co-founder, president and CEO of Transgeneron, tells TechJournal South the company is developing biotherapeutics that regenerate tissues, including the special cells that produce insulin. Type 1 diabetes is caused by loss of insulin-producing beta cells of the islets of Langerhans in the pancreas.

The technology evolved from work by company co-founder Li-jun Yang, M.D., an associate professor and pathologist at the University of Florida School of Medicine. Yang has worked for years on transdifferentation of cells—such as changing liver cells, which are very plastic, into beta-like insulin producing cells.

Yang, says Molony, discovered that one of the protein factors they used to reprogram the liver cells in the culture dish actually works as a therapeutic by itself. “You don’t have to take the cells out, reprogram them and re-inject them,” says Molony.

40 days later, normal mice
In animal studies, the company found that giving diabetic mice one injection a day of that protein for 10 days reduces their blood glucose (sugar) back to nearly normal. In 40 days after the last of the 10 injections, “The mice are completely normal,” says Molony.

“We don’t know how well this will work in a human. The whole purpose of the company is to work out what it will take to make this protein a therapeutic in man, the dose, and whether or not it will need other factors. It works by itself in mice.”

As in much bioscience, many questions remain, notes Molony. In Type 1 diabetes, the immune system destroys the Islet cells and they do not know what affect the immune system will have on the regenerated cells. No autoimmune reaction occurs after treatment in the mice experiments.

The company hopes the treatment will also help people suffering from Type II diabetes regain their ability to regulate glucose, says Molony.

Other master switches possible
She says later, the company hopes to validate other “master switches” to repair damaged heart or cardiovascular tissue. “We think that’s an area where there is a big need,” she adds.

The company is looking for seed capital of about $1 million, which would last it six months and get them working toward submitting an Investigational New Drug application to the U.S. Food and Drug Administration.

“Then we’d need to raise a larger round,” says Molony. It will likely take the company two years before it can get the therapeutic to the clinical trial stage, she notes.

In addition to Dr. Yang, the company’s board includes some familiar names: Dr. Dani P. Bolognesi, who retired as CEO of Research Triangle, NC-based Trimeris, in March; and Mark A. Cochran, a founding partner of NeuroVentures and Currently Chief CEO and executive director of the Blanchette Rockefeller Neurological Research Institute in Morgantown, WV.

On the Web: www.pathology.ufl.edu/~lyang/

www.neuroventures.com/thePeople.html

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