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Executive Summary

GENENTECH/GENVEC CYSTIC FIBROSIS GENE THERAPY COLLABORATION is the first corporate R&D deal for the newly formed private gene therapy firm GenVec, which will be headquartered in Montgomery County, Maryland in the metropolitan Washington, D.C. area. Under the agreement, Genentech will provide GenVec with up to $17 mil. in research support, milestone payments and future equity investments for cystic fibrosis (CF) therapy and for other serious diseases, Genentech announced March 3. The collaboration demonstrates Genentech's continuing interest in cystic fibrosis. The company plans to submit a PLA for Pulmozyme (DNase or dornase alfa) for improvement of lung function and reduction of respiratory infections in people with CF at the end of the first quarter. GenVec's initial focus will be on second-generation delivery systems for in vivo delivery of normal CF genes into patients with the disease. GenVec's scientific founder is Ronald Crystal, MD, chief of the National Heart, Lung & Blood Institute's Pulmonary Branch. On April 1, Crystal will move to Cornell Medical Center's New York Hospital as chief of the Division of Pulmonary and Critical Care Medicine. Crystal will work full-time for Cornell but will also be the head of GenVec's science advisory board and will be an NIH guest worker ("special volunteer") continuing to oversee his lab group and its first-generation cystic fibrosis gene therapy protocol, which was approved by NIH's Recombinant DNA Advisory Committee last December ("The Pink Sheet" Dec. 7, 1992, p. 15). The December-approved Crystal/NHLBI CF protocol is for a study in cystic fibrosis patients of a replication-deficient recombinant adenovirus (AdCFTR) to transfer the normal human CF transmembrane conductance regulatory (CFTR) cDNA directly to the respiratory epithelium of adults with cystic fibrosis. The first-generation, adenovirus-based protocol, which is awaiting FDA approval, could commence within the next six weeks to two months, Crystal said. His association with the NIH lab and project will be phased out through early 1994. GenVec received initial venture capital funding of $8.5 mil. from Hillman Medical Ventures. Hillman general partner Hal Broderson is acting as CEO for GenVec while the firm is engaged in a search for a permanent chief exec. By the end of 1993, GenVec hopes to have its permanent lab facilities set up with approximately 12 researchers on board and be in the clinic with a CF protocol. The next RAC meeting is scheduled for June 7-8. GenVec anticipates that its first CF protocol will go before the committee after the upcoming meeting. In addition to in vivo gene therapy for cystic fibrosis, GenVec will also look at other life-threatening diseases. The company projects having its first gene therapy for cancer in clinical trials in 1994.

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