Cystic Fibrosis Gene Therapy
Level - GradGenzyme Corporation is a biotechnology company with one of the largest gene therapy programs in the world. Genzyme’s gene therapy efforts began in 1991 when they began to develop a gene therapy for cystic fibrosis. Although the company has since expanded its gene therapy efforts to target cardiovascular disease, cancer, and lysosomal storage disorders, the development of an effective gene therapy for cystic fibrosis remains one of the company’s main gene therapy research goals.
Genyzme has just completed an extensive clinical trial in which the company tested the efficacy of its cystic fibrosis gene therapy protocols. The results of the trials suggest that gene therapy for cystic fibrosis may be feasible but that substantial improvements in the current protocols are needed before treatment will be effective (see references cited below). Based on these findings, Genzyme has decided to reevaluate whether or not the company should continue to work on developing a gene therapy for cystic fibrosis. Genzyme would like to know what is the greatest obstacle or rate-limiting step to achieving efficient gene transfer to the lung. Your determination should be based on an engineering analysis of the problem.
Resources:
Nael A. McCarty, Ph.D.
Associate Professor
School of Biology
Georgia Institute of Technology
310 Ferst Dr., Room 207 CE
Atlanta, GA 30332
nael.mccarty@biology.gatech.edu
404-385-2955 or 2956
Judith A. St. George, Ph.D.*
Director, Gene Therapy
Genzyme Corporation
Framingham, MA 01701-9322
judith.st.george@genzyme.com
Joe Le Doux – Room 2315, IBB
joe.ledoux@bme.gatech.edu
Manuscripts reporting the results of the latest Genzyme-sponsored clinical trials
1. Human Gene Therapy (2001) Jul 20; 12(11):1369-1382
2. Human Gene Therapy (2001) Jul 20; 12(11):1383-1394
*Available for a conference call