Brazil Joins US to Make Royalty-Free Drugs for Orphan Diseases

Fiocruz's equipment to produce vaccines Brazil's Oswaldo Cruz Foundation (Fiocruz) and Boston-based Genzyme Corp. have joined into a research collaboration pact to advance new treatments for certain neglected diseases. Fiocruz and Genzyme will focus initially on Chagas disease, a life-threatening infectious disease affecting millions of people in Latin America.

Fiocruz, a Brazilian public institution linked to Brazil's Ministry of Health, which promote health and social development, has expertise in biology, medicine, clinical treatment and the epidemiology of infectious disease.

Genzyme, on the other hand, is a leading global biotechnology company that is participating in efforts to discover and advance novel treatments for neglected diseases such as malaria and sleeping sickness through its Humanitarian Assistance for Neglected Diseases initiative.

Fiocruz and Genzyme will collaborate initially on two specific research programs. The first will focus on identifying new biological targets within the parasite that causes Chagas disease and will include screening for potential compounds that affect these targets and which could be developed into drugs.

The second program will test the effectiveness of using monoclonal antibodies to neutralize a protein that contributes to heart damage in Chagas disease, known as transforming growth factor-beta.

Under the partnership agreement, scientists from Fiocruz and Genzyme are expected to work in each other's laboratories from time to time. The collaboration includes an innovative approach to intellectual property rights, providing Fiocruz rights to commercial uses within the field of neglected disease on a royalty-free basis.

Both organizations bring strong capabilities to these research areas. Fiocruz is one of the world's leading centers for the study of Chagas disease, also known as American trypanosomiasis.

The disease is named for Carlos Chagas, the Brazilian physician who first discovered the disease, its cause and mode of transmission in 1909 while working at Fiocruz.

In addition, scientists at Fiocruz have developed metabolic maps of the Trypanosoma cruzi parasite that causes the disease when it is transmitted to humans from insects. These maps will be used to explore specific metabolic pathways that may serve as targets for potential drugs.

Genzyme has considerable experience in several of these pathways and is also a recognized leader in research on transforming growth factor-beta. In addition, Genzyme is developing and testing new compounds intended to treat African trypanosomiasis – a fatal infectious disease caused by a related parasite – in collaboration with the Drugs for Neglected Diseases Initiative.

"Neglected diseases pose significant problems in developing countries all over the world," said Paulo Buss, M.D., president of the Oswaldo Cruz Foundation. "In Brazil, with the support of the health ministry, we are accelerating our efforts to address these problems and to increase scientific activity in this area.

"We are very excited to partner with Genzyme, one of the world leaders in biotechnology, and we are optimistic that this collaboration will create promising opportunities that may help us deal with the burden of infectious disease."

Henri A. Termeer, chairman and chief executive officer of Genzyme Corp., said: "Biopharmaceutical companies have great potential to help address neglected diseases. Our industry possesses the precious capabilities to unlock the promise of the lab by converting basic science into drug candidates and increasing the number of potential treatments flowing into clinical development.

"We look forward to building a productive, sustainable partnership with Fiocruz, and we hope that this novel way of collaborating can serve as a model for public/private partnerships in the area of neglected disease drug development."

Rogerio Vivaldi, M.D., Genzyme's senior vice president and general manager for Latin America, commented: "This partnership with Fiocruz enables us to expand our support for research and development in Brazil and in Latin America, where we have already begun to increase our investment in clinical trials.

"Our work in neglected diseases is based on the same values and the same commitment to patients that have driven our work in developing treatments for people with orphan diseases."

Through its Humanitarian Assistance for Neglected Diseases initiative, Genzyme is participating in efforts to discover and advance novel treatments for neglected diseases. The initiative serves as a vehicle to identify, evaluate and manage scientific projects and partnerships focused on diseases that collectively affect hundreds of millions of people, including malaria, Chagas disease, sleeping sickness and other diseases.

The Oswaldo Cruz Foundation is the largest biomedical research institution in Latin America and one of the most respected in the world. Fiocruz forms part of the Brazilian Ministry of Health and plays an important role in the area of science and technology in health, including activities in basic and applied research, teaching, reference hospital and ambulatory assistance, strategies formulation in public health, information and diffusion, personnel training, vaccines, drugs, diagnostic kits and reagents production, quality control and development of technologies for health.

The organization is over 100 years old and was modeled on the Pasteur Institute from where Oswaldo Cruz (the Institute's founder and its first Director) drew inspiration.

Since 1981, Genzyme has grown from a small start-up to a diversified enterprise with more than 9,000 employees in locations spanning the globe and 2006 revenues of US$ 3.2 billion. Genzyme has been selected by Fortune as one of the "100 Best Companies to Work for" in the United States.

The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant, and diagnostic testing.

Genzyme established an affiliate organization in Brazil in 1997, becoming the first major biotechnology company to operate in the country. Over the past ten years, Genzyme has introduced in Brazil a number of treatments for rare inherited disorders (known also as "orphan diseases"), as well as treatments for kidney disease, thyroid cancer and organ transplant.

Genzyme employs more than 100 people in Brazil. The company's operations are based in Sao Paulo, and its office in Rio de Janeiro serves as a hub for commercial, medical, regulatory, and clinical research activities throughout Latin America.

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