Human Genome Sciences Inc., the Rockville-based gene discovery company, said yesterday that it will launch a new venture with St. Elizabeth's Medical Center in Boston to develop and market a promising gene therapy treatment for heart and other vascular diseases.
The new company, Vascular Genetics Inc., will focus on clinical development of a gene discovered by Human Genome, which the company found in laboratory studies and holds the promise for triggering growth of new blood vessels, opening up circulation in blocked areas.
Genes carry the DNA codes not only for characteristics of the body, but for many of its myriad functions.
The company is also being founded by Cato Holding, a Research Triangle, N.C.-based company that specializes in managing clinical drug trials, and Dr. Jeffrey Isner, a cardiologist at St. Elizabeth's. He invented the method for delivering the gene into muscle tissue.
"This company offers the quickest route to clinical development and clinical validation of this gene," said Dr. William Haseltine, chairman and chief executive officer of Human Genome.
Haseltine said the deal marked an important milestone for publicly held Human Genome, which has deals with several major pharmaceutical developers for access to its rich storehouse of genetic information.
"This is the first time we have unlocked the assets we have in gene therapy, and it could well prove a model for future deals," he said.
Yesterday, shares in Human Genome rose $1.31 to close at $42.31.
The new venture joins a growing list of companies seeking to develop gene therapy treatments for the vascular disease market.
Among the others: privately held GenVec of Rockville, which has a deal worth an estimated $100 million to jointly develop a gene therapy product to treat common cardiovascular diseases with Warner-Lambert's Parke-Davis pharmaceutical division; Genzyme Transgenics Corp. of Framingham, Mass.; and CaridoGene Therapeutics Inc. of Princeton, N.J.
At stake is a huge potential market. In the United States alone, there are 500,000 coronary bypass operations annually and more than 100,000 procedures to restore circulation to blocked limbs. An estimated 200,000 more are afflicted with peripheral vascular disorders, such as atherosclerosis.
The gene that Vascular Genetics will attempt to develop into a marketable treatment is known as vascular endothelial growth factor-2, or VEGF-2.
Haseltine said Human Genome's studies on the gene found that it is responsible for triggering the production of a naturally occurring protein responsible for blood vessel growth.
If fully developed and cleared for medical use, injections of the gene might prove useful in heart disease patients needing coronary bypass surgery or procedures to correct poor circulation caused by ulcers and disease.
"We have assembled a team with a combination of skills to get this developed in the shortest period of time possible," said Haseltine.
The announcement of the Vascular Genetics deal comes one day after Isner told heart disease experts attending a meeting of the American Heart Association in Orlando, Fla., that his team of researchers had seen dramatic results in a small group of heart disease patients treated with a similar gene to the one Human Genome discovered.
The treatment not only improved circulation in all but one patient, but reduced pain and enabled the patients greater freedom of movement, Isner reported.
Isner, who is also a professor at Tufts University School of Medicine, said that it is too early to tell whether the treatment could serve as a primary treatment for heart disease.
But he believes gene therapy for cardiovascular diseases could one day "become routinely available."
Under the agreement, Human Genome will provide Vascular Genetics with about $600,000 in start-up loan funds.
Human Genome holds 19.9 percent of the new company's stock, and warrants for the purchase of an additional 5.1 percent equity stake.
The company also has the option to buy all of the stock in Vascular Genetics, and would receive royalties on sales of any treatments commercialized.
Pub Date: 11/11/97