Thursday May 31, 10:24 am Eastern Time
Press Release
SOURCE: Genzyme Corp.
Genzyme Reports Progress in Gene Therapy Program
CAMBRIDGE, Mass., May 31 /PRNewswire/ -- Scientists from Genzyme Corp. will present 17 papers at the American Society of Gene Therapy's fourth annual meeting in Seattle this week. The presentations update progress made across the company's broad gene therapy research program, which is investigating potential treatments for genetic diseases, cardiovascular disease and cancer.
Seng Cheng, Ph.D., vice president for genetic diseases science at Genzyme, will review pre-clinical studies demonstrating the feasibility of gene therapy for Fabry disease, a lysosomal storage disorder that arises from the loss of the enzyme alpha-galactosidase. Genzyme has developed an enzyme replacement therapy for Fabry disease, Fabrazyme(TM) (agalsidase beta), which has received a positive opinion from the Committee for Proprietary Medicinal Products in Europe and is under review with the U.S. Food and Drug Administration. Dr. Cheng and his fellow Genzyme researchers have been able to demonstrate clearance of GL-3 lipid storage from affected tissues following gene transfer in pre-clinical studies of Fabry disease, an important ``proof of concept'' of the feasibility of using gene therapy to treat this disease.
This result was achieved with both adenovirus and adeno-associated virus (AAV) vectors, and the effect persisted for six months. AAV vectors, which Genzyme is exploring in a collaboration with Targeted Genetics, are particularly promising for treatment of lysosomal storage disorders such as Fabry disease, as they yield long term expression and appear safer than current-generation adenovirus vectors for the treatment of chronic diseases. These results are likely to be applicable to other storage disorders and will form the basis for a common approach to treating these diseases.
Other Genzyme scientists at the conference will provide progress updates in a number of areas, including the use of therapeutic angiogenesis to treat ischemic heart disease and peripheral vascular disease, inhibition of post- angioplasty restenosis, and the development of gene therapy for Gaucher disease. In addition, Genzyme scientists will present several advancements of the core vector technology underlying the company's gene therapy program. Genzyme launched its gene therapy program in 1991, and it has since grown to include more than 125 staff members with expertise in vector development, pre-clinical biology, clinical development, regulatory affairs, and manufacturing. Highlights of the program in each of Genzyme's divisions include:
Genzyme Biosurgery (Nasdaq: GZBX - news) is conducting clinical trials evaluating the use of its gene therapy product, hypoxia-inducible factor-1 (HIF-1 alpha) to treat patients with peripheral vascular disease. HIF-1 alpha is designed to promote angiogenesis -- the growth of blood vessels -- in tissue that has been deprived of oxygen. In addition, the division is conducting clinical trials of HIF-1 alpha on patients undergoing coronary artery bypass grafting surgery who have an area of the heart that is not suitable for surgical revascularization.
Genzyme Molecular Oncology (Nasdaq: GZMO - news) is conducting clinical trials using an adenoviral vector to deliver two melanoma antigens to target dendritic cells ex vivo. The division also has initiated a phase 1-2 trial of this same gene vaccine used in vivo. Because it is gene-based, this approach has the potential to produce a prolonged presentation of tumor antigens to the immune system, resulting in a more powerful immune response.
Genzyme General (Nasdaq: GENZ - news) is currently developing gene therapies for Gaucher disease and Fabry disease, and has previously conducted seven clinical trials in cystic fibrosis.
Genzyme Corp. is a biotechnology and health care products company that develops innovative products and services for major unmet medical needs.
This press release contains forward-looking statements based on management's current expectations, including without limitation statements about: the planned timing of presentations; the potential receipt of marketing approval and launch of Fabrazyme in Europe; the potential use of gene therapy to treat Fabry disease; the benefits of the use of adeno-associated virus vectors for treatment of lysosomal storage disorders and other disorders; potential additional indications for gene therapies; potential indications for HIF-1 alpha; and the potential ability of the gene therapy vaccine being tested by Genzyme Molecular Oncology to elicit an immune response. Actual results may materially differ due to numerous factors, including: the ability to successfully complete pre-clinical and clinical development of gene therapy products; enrollment rates for clinical trials; the actual safety and efficacy of gene therapy products; the actual timing and content of decisions made by the FDA, the European Commission and other regulatory authorities concerning Fabrazyme and other products; the FDA's response to the clarification and additional data provided by Genzyme related to the BLA for Fabrazyme; the ability to manufacture sufficient quantities of products for development and commercialization activities; Genzyme's ability to obtain, maintain and successfully enforce proprietary rights protection for its products; Genzyme's ability to obtain and maintain strategic collaborations and license agreements; market acceptance of gene therapy products; the extent and impact of governmental regulations and any guidelines that may be adopted by the National Institutes of Health concerning clinical trials involving gene therapy products; the competitive environment for gene therapies and other therapies for lysosomal storage diseases, cardiovascular disease and cancer; the risks and uncertainties described in reports filed by Genzyme with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation Exhibit 99.2 to Genzyme's 2000 Annual Report on Form 10-K. Genzyme General stock, Genzyme Biosurgery stock and Genzyme Molecular Oncology stock are each a series of common stock of Genzyme Corporation. Therefore, holders of each of these series of stock are subject to all of the risks and uncertainties described in the aforementioned reports.
Genzyme® is a registered trademark and Fabrazyme(TM) is a trademark of Genzyme Corporation. All rights are reserved.
Genzyme's releases are available on the World Wide Web at www.genzyme.com. They are also available from Genzyme's fax-on-demand service at 1-800-436-1443 within the United States or 1-201-521-1080 outside the United States.
SOURCE: Genzyme Corp. |
