NIH Selects Regeneron for Knockout Mouse Project

Regeneron Pharmaceuticals, Inc. has announced that it has been awarded a five-year grant from the National Institutes of Health as part of the NIH's Knockout Mouse Project.

The goal of the Knockout Mouse Project is to build a comprehensive and broadly available resource of knockout mice to accelerate the understanding of gene function and human diseases.

Regeneron will use its VelociGene^® technology to take aim at 3,500 of the difficult genes to target and which are not currently the focus of other large-scale knockout mouse programs.

Regeneron has also agreed to grant a limited license to a consortium of research institutions, the other major participants in the Knockout Mouse Project, to use components of Regeneron's VelociGene technology in the Knockout Mouse Project.

Regeneron will generate a collection of targeting vectors and targeted mouse embryonic stem cells (ES cells) which can be used to produce knockout mice.

These materials will be made widely available to academic researchers without charge.

Regeneron will receive a fee for each targeted ES cell line or targeting construct made by Regeneron or the research consortium and transferred to commercial entities.

VelociGene is Regeneron's proprietary technology for creating genetic modification in a mouse in a high-throughput manner.

VelociGene is one of a suite of inter-related and validated technology platforms that Regeneron has created to build and accelerate its therapeutic drug discovery and development programs.

The platforms also include VelocImmune^®, Regeneron's proprietary technology for creating fully human, therapeutic, monoclonal antibodies.

"This NIH grant is great news for Regeneron and the Lower Hudson Valley," said U.S. Senator Charles E. Schumer.

"The new federal funding will help propel Westchester to the forefront of breakthroughs in medical research, answering important questions about the function of the genes that make up our bodies."

"Regeneron's work will have the potential to further new medical treatments that will benefit New York and the entire country."

Knockout mice are laboratory mice in which researchers have deactivated, or "knocked out," a specific gene of interest by disrupting or replacing it with an artificial piece of DNA.

The loss of the gene leads to changes in the physical and/or biochemical characteristics of the mouse, such as appearance or behavior.

By identifying and studying these changes, researchers can determine the role of each gene in the mouse's physiology and development.

These mice can then be used to develop better models of human diseases, such as cancer, heart disease, neurological disorders, and diabetes.

Under the NIH grant, Regeneron will be entitled to receive a minimum of $17.4 million over a five-year period.

The Company will receive another $1 million to optimize its existing C57BL/6 ES cell line and its proprietary growth medium, both of which will be supplied to the research consortium for its use in the Knockout Mouse Project.

Regeneron will have the right to use, for any purpose, all materials generated by Regeneron and the research consortium.

"I am delighted NIH has decided to award this grant to Regeneron Pharmaceuticals as part of their Knockout Mouse Project," said Congresswoman Nita M. Lowey.

"These federal funds will allow researchers in the Lower Hudson Valley to explore medical advances never before imagined and answer some of the most important questions about the function of the human body."

"Regeneron's work will have the potential to dramatically advance medical treatments and help eradicate some of the world's most devastating diseases and conditions."

"We are pleased that the NIH has selected VelociGene, one of Regeneron's key technology platforms, for the advancement of the Knockout Mouse Project," said George D. Yancopoulos M.D., Ph.D., President of Regeneron Research Laboratories.

"VelociGene, when combined with our suite of inter-related technology platforms, particularly VelocImmune, is helping provide Regeneron with our next generation of therapeutic drug candidates."

"My congratulations to the Head of our VelociGene Division, David M. Valenzuela, Ph.D., and the rest of our team for developing these breakthrough technologies and leading our efforts to work with the NIH on this very important project."

"We believe this NIH initiative has the potential to build on the information generated by the genomic sequencing efforts led by the Human Genome Project, allowing it to be used to understand the function of individual genes and to develop new treatments for human diseases."