Genetic Therapy reaches research deal Calif. company to help make artificial virus


August 02, 1996|By Mark Guidera | Mark Guidera,SUN STAFF

Genetic Therapy Inc., the Gaithersburg biotechnology company attempting to develop ways to treat diseases with genes, is teaming up with a California company to develop an artificial virus as a way to carry genes directly to cells to control or block diseases.

The collaboration between Genetic Therapy and Advanced Therapies Inc., a small privately held biotechnology company in Novato, Calif., is worth about $11 million in research and other payments to Advanced Therapies, the companies said.

Genetic Therapy, which was bought by Swiss drug giant Sandoz AG last year, also would pay the California company royalties if any products are developed and approved for use from the research, Advanced Therapies said.

Specifically, Genetic Therapy will pay Advanced Therapies for the rights to help develop its promising new type of "vector," or delivery device, for gene therapy.

The new vector has shown promise in the laboratory for treating cancer and pulmonary disease, said Hans Schreier, chief

scientist at Advanced Therapies who developed the technology while he was a researcher at the University of Florida.

Schreier said he and researchers at Advanced Therapies have been working for about two years on creating the vector, called the Artificial Viral Envelope, or AVE.

It's based on a merger of the structure and properties of viruses and those of liposomes, which are microscopic sacs made of fats that are often used in cell research.

The AVE acts like a virus in that it can recognize and enter specific cells "with high selectivity and efficiency," said Schreier.

But AVEs lack the actual viral capabilities of a virus and therefore don't trigger inflammations or other ill side-effects.

"These are really new entities that combine the useful aspects of viruses, but don't have the bad side effects other gene vector researchers have been struggling with," said Schreier.

"It's a whole new paradigm."

He said AVEs are easily made in the lab and can be administered to transport drugs, proteins or enzymes, as well as genes, to specific disease sites.

Schreier said the companies believe AVEs can be loaded with the right genes, which contain proteins that tell cells what to do, and inject them in the body so they head for a disease site, such as a tumor.

The hope would be that once at a diseased site, AVEs would release the genes, which in turn would show cells how to fight or block disease.

Schreier said the companies hope to launch animal trials with AVEs late this year at Genetic Therapy's Gaithersburg labs. Clinical trials on humans would be at least several years away, he said.

No one has shown yet in human trials that gene therapy is an effective way to treat diseases such as cancer. But numerous biotechnology and pharmaceutical companies have invested millions of dollars in developing a genetic therapy and proving it effective.

Many leading researchers believe gene therapy will one day emerge as an effective way to treat at least some illnesses.

The market value for such a way to treat disease is still unclear, say analysts, but it could be quite large if the therapy is shown to be effective with minimal side effects.

Pub Date: 8/02/96

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