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Three of a kind
March 2010
by David Hutton  |  Email the author
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BASEL, Switzerland—In an effort to step up its stem cell research activities, Roche has inked a deal with Massachusetts General Hospital (MGH) and Harvard University to use stem cell technologies to advance drug discovery in areas of high unmet medical need.
The aim is to develop cellular models of disease based on human stem cell lines and to investigate the potential efficacy, safety and toxicology profile of new drug candidates from Roche's compound library.

Cell lines will be derived from the tissues of healthy volunteers and from patients with various diseases.

Matthias Steger, Roche's global licensing director for Stem Cell Alliances, says Roche chose MGH and Harvard after an extensive landscape review of more than 20 academic institutions and biotech companies.

"The main reason we chose MGH/Harvard is because they are at the forefront of iPS reprogramming research, as well as having broad patient access through MGH," he says.
Under the terms of the agreement, the parties will collaborate across a broad range of disease areas, with an initial focus on metabolic disorders and cardiovascular disease. Roche will gain access to cell lines, protocols, data and materials in exchange for research funding over three to five years, with clinical development milestone payments for drug candidates discovered through stem cell disease models.

"We 're investing in the range of $20 million to $25 million over five years, depending on the outcome of currently designed research activities," adds Steger. "Roche will pay development milestones on the discovered compounds to MGH/Harvard."

A staff exchange program also will promote sharing of expertise between the three partners and a joint governance committee will oversee research progress.

Jean-Jacques Garaud, global head of Roche Pharma Research and Early Development, points out that as one of the most active pharmaceutical companies in this field, Roche will work with MGH and Harvard to "ensure that all our leading experts bring the potential of stem cell research to fruition."

"This technology is like having a disease in a test tube and being able to test possible effects of drugs on 'virtual' patients—translational medicine at its best," he notes.

Dr. Kenneth Chien, director of the MGH Cardiovascular Research Center, says the collaboration "captures that potential at the interface of academia and biotechnology."
"The ability to generate cellular models of human cardiovascular disorders from human pluripotent stem cells is starting to revolutionize our approach to the discovery of new disease pathways and therapeutic strategies," he says.

The collaboration also taps into myriad possibilities presented by stem cell research, according to Doug Melton, co-chairman of Harvard University's Department of Stem Cell and Regenerative Biology.

"Stem cells are powerful tools for discovering new medical treatments and bridging the gap between the laboratory and the clinic," he notes. "This collaboration will use our pioneering research to help deliver new ways of improving the lives of patients suffering from diseases that cannot currently be addressed."

Roche has forged a number of collaborations focused on evaluating stem cell-based approaches for drug discovery. In June 2009, the firm signed a $10.36 million, two-year collaboration with the Institute for Stem cell Therapy and Exploration of Monogenic diseases (I-STEM) focused on the use of I-STEM's neuronal stem cell proliferation technologies in the screening of Roche's compounds for potential new candidates against neurodegenerative diseases.

In 2008, Roche partnered with U.K.-based stem cell consortium Stem Cells 4 Safer Medicines (SC4SM) to generate a repository of stem cells suitable for toxicology testing in high-throughput platforms. The initiative is being fund primarily by the U.K. government, with Roche and two other pharmaceutical companies also contributing. During the same year, the firm signed an agreement with Cellular Dynamics to test a number of its drug compounds for cardiotoxicity.



Roche, Genentech license flu-targeting antibodies from Dana-Farber and Sanford-Burnham Institute

BASEL, Switzerland—Roche and its wholly owned group member Genentech have signed a license agreement with the Dana-Farber Cancer Institute and the Sanford-Burnham Medical Research Institute that grants the companies exclusive rights to manufacture, develop, and market human monoclonal antibodies to treat and protect against group 1 influenza viruses.

Roche and Genentech, who announced the agreement Feb. 8, also have a non-exclusive right to manufacture, develop and market diagnostic tests for group 1 influenza. Complete terms of the agreement are not public, but Dana-Farber and Sanford-Burnham will receive license fees and may receive milestone payments and royalties.

The viruses include the strains for the current seasonal and H1N1 influenzas. The discovery of the antibodies was first reported by Dr. Wayne A. Marasco, associate professor of medicine at Dana-Farber and Harvard Medical School; Dr. Robert Liddington, professor and director of the Infectious and Inflammatory Disease Center at Sanford-Burnham; and Dr. Ruben Donis, chief of the Molecular Virology and Vaccines Branch at the Centers for Disease Control and Prevention, in Nature Structural and Molecular Biology in February 2009.

The researchers demonstrated that the newly identified antibodies attach to the stem region of the viral proteins (hemagglutinin), rather than to the head region, the standard target of current influenza vaccines. According to the researchers' report, binding to the highly conserved stem region prevents changes in the protein that are necessary for viral entry into the host cell, thereby inhibiting further infection of host cells and the rise of escape mutants.

The researchers also noted that standard influenza vaccines that consist of an attenuated, or killed, virus typically stimulate antibodies against the protein's head. These vaccines are less effective as the head region is prone to change, leading to the rise of forms of the virus that can evade neutralizing antibodies.
 
 
Code: E031001

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