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Genzyme 2004 Annual Report
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Table of Contents
2004 - A Transforming Year
Financial Highlights
Letter To Shareholders
A Future of Hope
Building a Sustainable Future
Focused Medical Areas
Genetic Diseases
Renal Disease
Orthopaedics
Oncology
Transplant / Immune Diseases
Genetics / Diagnostics
A Future of Innovation
Late-Stage Clinical Programs
Progress Across Medical Areas
Genetics / Diagnostics Pipeline
Product Development Pipeline
A Future of Commitment
A Future of Growth
A Future of Innovation
Genzyme's sustainability depends upon constant innovation and discipline in the drug discovery and development process. We take a portfolio approach, focusing development efforts on specific medical areas and selecting from a broad technology base to identify the best treatment alternative for a particular disease.

Science at the core
Genzyme has built its research and development organization on scientific thinking and practices of the highest quality, and we have been honored for the second consecutive year as a top employer of scientists on a global basis in a survey by the American Association for the Advancement of Science. As well as the excellence of our science, it is the way our discovery and development efforts are organized that makes us productive.

To help manage the risk inherent in the drug discovery process, we have built a broad array of technology platforms — including proteins, small molecules, polymers, biomaterials, and gene and cell therapies. Even so, we do not look at biological problems from a technology perspective. Rather, we focus on a diverse set of five medical areas aligned with our business units. Within these areas, we pursue a variety of diseases and identify the most promising technologies. Through this approach, we have discovered molecules that show promise for multiple diseases or for working in combination with other therapies. We have also been able to target new indications for existing products by fully investigating mechanisms of action.

Pipeline management
Another key strategy for risk reduction is managing the pipeline so that it contains a healthy balance of products at all stages of development. To accomplish this objective, we seek product candidates both from our own laboratories and through collaborations of various types. Our scientific expertise allows us to thoroughly evaluate partnership options in order to identify the most promising opportunities for us to make a contribution to product development.

Global resources
In research and development, as in other aspects of business, Genzyme is expanding its geographic horizons. In early 2004, we opened our new discovery research center in Cambridge, United Kingdom, a world hub for biotechnology. Our work there is concentrated on antibody technology and its applications in oncology, renal disease, and immune-mediated diseases, and exciting possibilities are already emerging. This global scientific approach mirrors that taken by our well-established and successful clinical and regulatory organization. Clinical and regulatory hubs are located in the United Kingdom, the Netherlands, and Japan, allowing us to move our products to the market rapidly on a broad front.

In the United States, we are also making significant investments in research infrastructure. We are expanding our facility for small molecule and polymer research in Waltham, Massachusetts, and adding 120,000 square feet of dedicated research space on our main research campus in Framingham, Massachusetts. Both U.S. facilities are expected to open in 2006.

Micrograph of a Niemann Pick Liver

Genzyme is planning to initiate a clinical trial for ASM-deficient Niemann Pick disease. This microscopic image of a Niemann Pick liver is stained in red with CD68 to highlight Kupffer cells enlarged with sphingomyelin, a central component of the disease.

Micrograph of Thyrogen structure

This microscopic image reveals the structure of Thyrogen, our thyroid cancer dignostic product. We are investigating the use of varied formulations of thyroid-stimulating hormone (TSH) for diseases ranging from multinodular goiter to osteoporosis.