Lysosomal Storage Disorders

These inherited diseases, which typically affect fewer than 10,000 people worldwide, are caused by enzyme deficiencies. Genzyme markets four enzyme replacement products to treat these diseases, which can be life-threatening.

Cerezyme^® (imiglucerase for injection) – for Type 1 Gaucher disease.

Fabrazyme^® (agalsidase beta) – for Fabry disease.

Aldurazyme^® (laronidase) – for Mucopolysaccharidosis I.

Myozyme^® (alglucosidase alfa) – for Pompe disease.

Renal Disease

Renagel^® (sevelamer hydrochloride) is Genzyme’s calcium-free, metal-free phosphate binder that reduces phosphorous levels in chronic kidney disease patients who are on dialysis.

Renvela^® (sevelamer carbonate) is a next-generation version of Renagel, currently approved in the U.S. for use in dialysis patients. Genzyme is pursuing worldwide approvals and seeking to expand Renvela's indication to include patients with earlier stages of chronic kidney disease.

Hectorol^® (doxercalciferol) is a vitamin D₂ treatment for secondary hyperparathyroidism in chronic kidney disease patients.

Orthopaedics / Biosurgical Specialties

Synvisc^®, Synvisc-One^™ (hylan G-F 20) Synvisc is one of the world’s top therapies to treat the pain of osteoarthritis of the knee; Synvisc-One offers a new, single-injection treatment option.

Carticel^® (autologous cultured chondrocytes) is a cell-based therapy to help repair damaged knee cartilage currently marketed in the U.S.

MACI^® (Matrix-induced Autologous Chondrocyte Implantation) is a cell-based therapy to help repair damaged knee cartilage currently marketed in Europe and Australia.

Seprafilm^® is designed to prevent painful and potentially debilitating adhesions following surgical procedures.

Epicel^® (cultured epidermal autografts) is a cell-based therapy for treating patients with severe, life-threatening burns.

Transplant and Immune Diseases

Thymoglobulin^® (anti-thymocyte globulin, rabbit) treats acute rejection in kidney transplant patients by suppressing the body’s natural immune response. It is also being studied for liver transplant.

Genzyme has several products in development for immune diseases such as multiple sclerosis and pulmonary fibrosis.

Oncology

Campath^® / Mabcampath^® (alemtuzumab), is approved in the U.S. and Europe as a first-line treatment for B-cell chronic lymphocytic leukemia (B-CLL).

Clolar^® / Evoltra^® (clofarabine), is approved in the U.S. and Europe for the treatment of children with refractory or relapsed acute lymphoblastic leukemia.

Thyrogen^® (thyrotropin alfa for injection), is used in thyroid cancer follow-up, allowing patients to be screened without having to suspend thyroid hormone therapy. It is also approved in the U.S. and Europe for use in remnant ablation, a procedure patients commonly undergo when being treated for thyroid cancer.

Genetics / Diagnostics

Genzyme is one of the largest providers of diagnostic testing services in the world, with expertise in reproductive and oncology testing. Genzyme is also a major supplier of point-of-care diagnostic products, with a focus on infectious diseases, diabetes and cardiovascular disease.

The company is playing an important role in the movement toward personalized medicine by developing tests that can help physicians identify how patients are likely to respond to targeted therapies.

Research and Development

Genzyme's research and development efforts are focused on the areas of medicine where it markets commercial products. The company also conducts research in cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need. In 2007, Genzyme invested approximately $640 million in this work, or about 17 percent of its revenues.

Genzyme continues to build its pipeline through both internal research and licensing and acquisitions, focusing on product candidates with the potential to change the standard of care for serious diseases. The company’s pipeline is concentrated on programs in mid- to late-stage development and includes approximately 25 phase 2 studies and several major pivotal studies.

Therapies in late-stage development include: alemtuzumab for multiple sclerosis; Mozobil^™(plerixafor), which is designed to improve the outcome of stem cell transplantation in patients with blood cancers; Clolar, for adult acute myeloid leukemia; and mipomersen for familial hypercholesterolemia and other high risk, high cholesterol patients.

Corporate Responsibility

Corporate responsibility is a priority at Genzyme. The company’s commitment to patients extends beyond the development of new treatments and the services needed to deliver them. Genzyme has a strong presence in developing countries, where it provides free medicine to patients and helps to build sustainable health care systems. The company in 2006 launched an initiative to support the development of treatments for neglected diseases that affect hundred of millions of people in the developing world. Genzyme’s headquarters is one of the most environmentally responsible office buildings in the world, and the company is a leader in waste-reduction and recycling efforts.

In addition, Genzyme supports science education and health initiatives in the communities in which it operates. For these and other efforts, Genzyme has been included for the past three years in the Dow Jones Sustainability World Index, which consists of companies that excel in economic, environmental and social performance. In 2007, Genzyme was named one of the "Global 100 Most Sustainable Corporations in the World" by Innovest Strategic Value Advisors. BusinessWeek has ranked Genzyme as one of the top corporate givers, and the company has also been recognized by the U.S. Environmental Protection Agency, the American Association for the Advancement of Science, and many other organizations.