A GUIDE TO BIOMANUFACTURING AT GENZYME

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1 M A K I N G P R O T E I N T H E R A P I E S A GUIDE TO BIOMANUFACTURING AT GENZYME

2 Genzyme strives to make a life-changing difference for patients with serious diseases by discovering, developing, and Making Protein Therapies Compared to the automated manufacturing of chemical drugs, which can take days, the production of therapeutic proteins is a complex, technology-based process that requires months. manufacturing innovative therapies. The way we make our protein therapies I T B E G I N S W I T H A C E L L is called biomanufacturing, which uses living cells. At our biomanufacturing facilities around the world, we use living cells to produce human proteins that are missing or not working properly in people with often life-threatening diseases. The biomanufacturing process begins when a piece of human DNA is inserted into a living cell so that the cell can produce the human protein. Cells are frozen and stored in a cell bank. When they are needed, they are removed from the cell bank, thawed, and mixed with a nutrient-rich solution called growth medium, in which they grow and reproduce. Over a period of weeks, the cells will multiply from several million to more than 30 billion. Each cell produces a small amount of the protein that will eventually be used in the therapy. *Approximate number of days into the manufacturing process. Small-scale bioreactor containing growth medium and production cells.

3 In the production bioreactor, the cells grow and continue to release the protein into the growth medium. Every day, samples of the liquid in the bioreactor are tested to monitor the growth and quality of the cells. The harvest process regularly removes the protein from the bioreactor, while the cells stay behind to produce more protein. H A R V E S T I N G P R O T E I N B I L L I O N S O F C E L L S These billions of cells are transferred to a bioreactor, which is a sophisticated container that monitors and controls the cells environment, including temperature, oxygen level, mixing rate, ph, and more. Over the next several weeks, as the cells continue to multiply, they are transferred to much larger bioreactors, until there are enough cells for the final production bioreactor. Final production bioreactor, which can stretch 3 stories high. The tops of the stainless-steel harvest tanks in the manufacturing suite.

4 R E A D Y F O R D E L I V E R Y The harvested material, which contains other substances along with the needed protein, is put through a series of filters to eliminate unwanted compounds. Each batch undergoes strict quality control to ensure purity and safety. A single dose of medicine consists of several milligrams of the purified protein, which requires billions or trillions of cells to produce. P U R I F Y I N G T H E M E D I C I N E The purified protein solution is filled into small vials for distribution. This is performed in a sterile environment to prevent contamination and ensure safety. Some therapies also go through a freeze-dry process. At the finishing stage, each vial is inspected individually for irregularities. Samples are taken through a final quality control to ensure product purity, safety, and potency. The vials of finished drug are labeled and packed before they are shipped. Some of the column filters used in the purification process. An employee performing one of several tests in the manufacturing process.

5 Patients receive protein therapy by intravenous (IV) infusion. The harvested material, which contains other substances along with the needed protein, is put through a series of filters to eliminate unwanted compounds. Each batch undergoes strict quality control to ensure purity and safety. A single dose of medicine consists of several milligrams of purified protein, which requires billions or trillions of cells to produce. PURIFYING THE MEDICINE M E E T I N G PAT I E N T N E E D S The vials of protein therapy reach their final destinations, providing treatment and restoring hope to patients around the world. Genzyme is committed to the patients and families who rely on our products. We are proud that the processes and medicines pioneered here bring new hope to thousands of patients suffering from serious diseases. LARGE-SCALE GROWTH CELL PREPARATION SMALL-SCALE GROWTH Some of the column filters used in the purification process. PURIFICATION PRODUCTION AND HARVEST FILL AND FINISH

6 One of the world s leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with approximately 10,000 employees. Genzyme operates biomanufacturing facilities around the world and helps patients in over 100 countries. Genzyme Corporation 500 Kendall Street, Cambridge, MA T F This brochure was prepared for informational purposes only. The general production process and timelines depicted in this brochure do not necessarily represent the actual production process or timeline used by Genzyme Corporation or its affiliates, partners, or contractors for any of its products Genzyme Corporation Genzyme is a registered trademark of Genzyme Corporation. All rights reserved. LSD-US-P