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Genzyme Launches Initiative To Assist the Development of Treatments for Neglected Diseases

Genzyme Launches Initiative To Assist the Development of Treatments for Neglected Diseases

Published 04-10-06

Submitted by Genzyme Corporation

CAMBRIDGE, Mass. - Genzyme Corp. (Nasdaq: GENZ) today announced that it has established a program to participate in efforts to discover and advance novel treatments for neglected diseases affecting the developing world. The company's new Humanitarian Assistance for Neglected Diseases initiative (HAND) will serve as a vehicle to identify, evaluate and manage scientific projects and partnerships focused on diseases that collectively affect hundreds of millions of people. These could include malaria, tuberculosis, leishmaniasis, Chagas disease, sleeping sickness and other diseases.

Genzyme will focus on projects where it can play a defined role in the process of moving potential new treatments from discovery toward clinical testing. The company will not seek to profit from the commercialization of any products it helps to develop. It will grant all commercial and intellectual property rights in neglected disease areas to non-profit partners. The HAND initiative complements existing Genzyme programs that provide free medicines and help to build sustainable health care systems in developing countries.

Genzyme Chairman and Chief Executive Officer Henri A. Termeer made today's announcement at the annual meeting of the Biotechnology Industry Organization in Chicago.

"Society has a responsibility to do more to reduce the devastating impact of infectious disease in the developing world," said Mr. Terrmeer. "Industry has a unique contribution to make by applying drug discovery and translational research capabilities to create new solutions--filling a gap where there is clearly a great need. As a single company, we can play only a small part. But we hope to act as a catalyst to encourage the involvement of others by creating partnership models that demonstrate how companies can contribute in ways that shareholders will support and other important stakeholders will welcome."

Mr. Termeer said that Genzyme hoped to play a leadership role in increasing activity in the area of neglected diseases as it has in the field of orphan diseases. Genzyme's creation of a sustainable model for developing treatments for orphan diseases has helped encourage other companies to allocate resources to an area that previously had been neglected.

"Progress will come when it is acceptable for companies to devote some portion of their resources to treatments for diseases affecting people in the developing world," added Mr. Termeer. "This is a long-term project, but I hope that in ten years we can say this is becoming a more common way to do business throughout the biotechnology and pharmaceutical industry."

Genzyme has begun one of its first projects in partnership with the Drugs for Neglected Diseases initiative (DNDi), a not-for-profit drug development organization created to address the critical lack of needs-driven drug research and development for neglected diseases. Genzyme and DNDi are working to develop and test novel compounds intended to treat African trypanosomiasis, more commonly known as sleeping sickness, a fatal infectious disease that has grown to epidemic proportions in sub-Saharan Africa. Some testing under this agreement will also be done through an agreement with the Swiss Tropical Institute.

The project will focus initially on seeking novel treatments that target the same biochemical pathways as those targeted by eflornithine. Eflornithine is the most effective drug for treating the central nervous system manifestations of the disease, but it must be given as an infusion four times a day at very high doses, a regimen that is not practical in those areas where the disease is endemic. Genzyme's expertise in research on eflornithine and polyamine pathways for treatment of cancer may allow the company to contribute to this effort. Genzyme acquired certain rights to eflornithine, also known as DFMO, through its purchase of Ilex Oncology Inc.

Founded by six public-sector research institutions and an international humanitarian organization, DNDi was formed in 2003 to develop drugs for the most neglected diseases by promoting a needs-driven R&D agenda. It works to fill the existing research and development gaps in drugs for neglected diseases by initiating and coordinating projects in collaboration with the international research community, the public sector, the pharmaceutical industry and other relevant partners, and through advocating for greater public responsibility and leadership in addressing the needs of patients with neglected diseases.

Genzyme also has begun collaborating with the Broad Institute of MIT and Harvard to discover and advance new therapeutic candidates for malaria, a devastating disease caused by a mosquito-borne parasite. Approximately 300-500 million cases of malaria occur each year, killing more than one million people annually, with young children and pregnant women being the most vulnerable. The majority of malaria cases occur in sub-Saharan Africa, but the disease also remains a significant health problem in Asia and South America.

"For the first time in history we have the tools and the expertise to truly understand how malaria works," said Eric Lander, director of the Broad Institute. "This scientific collaboration with our Genzyme colleagues can take us to the next step of finding effective treatments."

Together with the Broad Institute, Genzyme has entered into a memorandum of understanding with Medicines for Malaria Venture (MMV), a public-private partnership working to develop new and better drugs for malaria. MMV will provide clinical guidance, project management and coordination with other anti-malarial drug-development efforts, and will work with other parties to secure sources of funding for further work.

"This collaboration with Genzyme and the Broad Institute will significantly enhance MMV's drug discovery capacity and further boost our ability to develop completely new anti-malarials," said Dr. Chris Hentschel, president and CEO of MMV. "Innovative partnerships like these are changing the landscape of research and development for neglected diseases."

Genzyme's Social Responsibility Programs
Genzyme has a strong presence in developing countries, where it provides free medicine and helps to build sustainable health care systems. Among the programs Genzyme sponsors is the Gaucher Initiative, a partnership with Project HOPE that brings Cerezyme® (imiglucerase for injection) therapy to patients with Gaucher disease who live in developing countries where reimbursement is not yet a reality. The Gaucher Initiative, established in 1999, provides treatment to patients and increases understanding of Gaucher disease among physicians, patient advocates, and governmental organizations. Genzyme continues to expand the program and build on its model to create new programs for patients receiving other products. For more information about the Gaucher Initiative and similar Genzyme programs, please visit the Our Commitment to Patients section on the company's Web site: http://www.genzyme.com/commitment/patients/patients_home.asp

About Genzyme
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. This year marks the 25th anniversary of Genzyme's founding. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 8,000 employees in locations spanning the globe and 2005 revenues of $2.7 billion. Genzyme has been selected by FORTUNE as one of the "100 Best Companies to Work for" in the United States.

With many established products and services helping patients in more than 80 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune diseases, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as heart disease and other areas of unmet medical need.

This press release contains forward-looking statements regarding Genzyme's HAND Initiative. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected, including that Genzyme's HAND Initiative is unsuccessful in identifying, evaluating and/or manage scientific projects and partnerships focused on diseases effecting large populations in developing nations. Please refer to the risks and uncertainties described in reports filed by Genzyme with the Securities and Exchange Commission under the heading "Factors Affecting Future Operating Results" in the Management's Discussion and Analysis of Financial Condition and Results of Operations section of Genzyme's Annual Report on Form 10-K for the year ended December 31, 2005 for a more complete discussion of the risks associated with Genzyme's business. Genzyme cautions investors not to place substantial reliance on the forward-looking statement contained in this press release. This statement speaks only as of the date of this press release, and Genzyme undertakes no obligation to update or revise the statement.

Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.

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Genzyme's press releases and other company information are available at www.genzyme.com and by calling Genzyme's investor information line at 1-800-905-4369 within the United States or 1-703-797-1866 outside the United States.

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Genzyme Corporation

Genzyme Corporation

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. This year marks the 25th anniversary of Genzyme's founding. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 8,000 employees in locations spanning the globe and 2005 revenues of $2.7 billion. Genzyme has been selected by FORTUNE as one of the "100 Best Companies to Work for" in the United States. With many established products and services helping patients in more than 80 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune diseases, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as heart disease and other areas of unmet medical need.

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