GeneMedicine Inc. and Vanderbilt University entered into a researchagreement that is expected to result in the Houston company's firstclinical trial. The alliance also starts "a major new initiative" byGeneMedicine into pulmonary gene therapy.
The cooperative research agreement has two parts. One part of thedeal calls for GeneMedicine to provide the Nashville, Tenn.,university's Center for Lung Research undisclosed pulmonary-related research funding in areas outside the scope of a trial they'llbe working on. That is a three-year agreement with a two-yearoption, Fred Ledley, GeneMedicine's founder and vice president offor clinical development, told BioWorld.
The second area involves a pulmonary gene therapy trial in whichthe center's director, Kenneth Brigham, will be principalinvestigator. The trial has received approval from the RecombinantAdvisory Committee of the National Institutes of Health, Ledleysaid, but not FDA approval.
GeneMedicine will supply plasmid DNA and gene delivery systemsfor the trial, which was proposed by Vanderbilt, Ledley said.GeneMedicine will get rights to gene therapy technologies resultingfrom the Center for Lung Research's work.
"We think the lung is a very important target for gene medicine,"Ledley said. "We think there will be a series of opportunities to treatlung disease and use the lung as a source of therapeutic proteins."
The delivery system being provided by GeneMedicine consistsprimarily of its proprietary cationic lipid, Dotma
Researchers first will attempt to express the alpha-1 antitrypsin genein volunteers who have deficiencies, which cause emphysema. "Thefirst part is to show we can get expression in the nose," Ledley said."The second part is to instill the DNA into the lobe of the lung forpatients who are going to have part of their lung removed."
Dotma is the prototype cationic lipid for gene transfer that has beenthe standard reagent in vitro for years, Ledley said. Brigham, as earlyas 1989, showed it also was useful in vivo for gene delivery to thelungs, he said.
GeneMedicine gained access to the Dotma technology through abroad collaboration agreement with Syntex (U.S.A.) Inc. a subsidiaryof Palo Alto-Calif.-based Syntex Corp. (since acquired by RocheHolding Ltd.), of Palo Alto, Calif., in April. GeneMedicine obtaineda co-exclusive worldwide license with an option to sublicense. (SeeBioWorld Today, April 15, 1994, p.1.) n
-- Jim Shrine
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