CHAPEL HILL, N.C. …Â Asklepios, a recent University of North Carolina biotechnology spinout, has hired former Bayer executive Alison Arter as chief executive officer and has signed a licensing agreement with UNC for its new gene-delivery technology.
Named after the Greek god of medicine, the company is based on the viral gene-delivery research of founder R. Jude Samulski, a Ph.D. in virology and director of UNC’s gene therapy center, and his colleagues. The research was funded by a total of about $12 million in National Institutes of Health grants.
Samulski says the new gene delivery method evolved from 20 years of study of the only human DNA virus that is non-pathological. Although people are exposed to the virus when they’re about 2 years old, they don’t get sick. Called the adeno-associated virus (rAAV), it was found in the 1960s when researchers sought the infectious agents causing colds and other diseases.
Gene keeps on giving
“We’ve since found it has a unique biology for gene delivery,” Samulski says. In addition to being non-harmful, he says, “It enters a cell and persists for the life of the cell. It is there permanently. We have data at UNC where we put this vector into a hemophiliac dog, and they’re still expressing the gene (it carried) after three and a half years.”
“One of the primary requirements of a successful gene delivery therapy … in addition to doing no harm … is its ability to persist,” he says. He notes that UNC researchers have modified the virus in ways “we think make it even more likely to succeed.”
The UNC researchers discovered they could desiccate the virus and reconstitute it. This stability is an extremely attractive quality in a gene-therapy reagent. Samulski points out that one of the reasons medicine eradicated smallpox is because its vaccine can be powdered, while polio persists, especially in Third World countries, because its vaccine must be frozen.
They have also found that the dried form can be applied with soluble sutures and deliver a needed gene exactly where needed. “This suggests you could apply them to biomedical devices such as stents used in arteries,” he says. Used during artery-opening surgery, they might deliver a gene that helps prevent further artery clogging.
Facing up to disease
Samulski says the researchers can change the face of the virus like changing the colored tiles of a Rubic’s Cube. That means it can be designed to enter a variety of different cells.
Asklepios has a mature patent portfolio already, and other patents are still in process, Arter says. “The number of diseases that could be tackled with this are very large. We’ll tackle a few and sub-license rights to others in specific areas,” she says.
The company plans to pursue two therapeutic projects itself initially: one for Duchenne’s muscular dystrophy and another for delivering genes to muscle, where the UNC lab research has already shown results.
Arter says the company plans to seek venture capital down the road. Eventually, Samulski says, it would require $4 million to $8 million to conduct a clinical trial of a treatment for a muscle disorder.
“We won’t need that for a while,” Arter says. In addition to NIH funding, she says the company plans to seek backing from the Muscular Dystrophy Association for its work on that disease.
The UNC gene therapy team has already successfully used the rAAV as a vector to carry a gene to two people with Canavan’s Disease. That neurological disease, caused by an enzyme deficiency, destroys the white matter of brain tissue.
Arter, who was vice president of commercial development at Bayer Biologicals Division, had worldwide responsibility for products from clinical development through marketing, sales, and follow-up. Her group was responsible for products making $1.2 billion in annual revenue, including those made in Clayton, N.C.
She says the four-employee Asklepios is currently virtual and is not actively looking for office space. “We might benefit from a stay in an incubator. We won’t be making anyone’s real estate day right away. But we’re off and running.”