NIH selects Regeneron for knockout mouse project

STORY UPDATE

HOUSTON—May 17, 2007—The Texas Institute for Genomic Medicine announced it had been invited to join the International Knockout Mouse Consortium, a network of organizations that includes the NIH's Knockout Mouse Project. The institute is already home to the world's largest collection of more than 200,000 mouse embryonic stem cell lines and expects to increase this number to 350,000 by year end.

TARRYTOWN, N.Y.—The National Institutes of Health (NIH) recently awarded Regeneron Pharmaceuticals Inc. a five-year grant as part of the NIH's Knockout Mouse Project, the goal of which is to build a comprehensive and broadly available resource of knockout mice to accelerate the understanding of gene function and human diseases.

Although there are others involved in the project—specifically a consortium of three academic institutions—Regeneron is playing a primary role, says Dr. George D. Yancopoulos, president of Regeneron Research Laboratories. Not only is Regeneron using its VelociGene technology to take aim at 3,500 of the most difficult genes to target, but it also is granting a limited license to the research consortium to use that same technology for its work.

"Our proprietary technology is really at the heart of this," Yancopoulos says. "And not only do we have the means to create any genetic modification in a mouse in a precise and high-throughput manner with VelociGene, we have also invested heavily in downstream technologies—VelociMouse and VelocImmune—to turn cells into mice rapidly and to create fully human monoclonal antibodies against targets we want to more fully explore. So we are really in a position to be at the forefront of this effort and have a significant pipeline generator."

Regeneron's work for the NIH projects revolves around genes that are not only hard to target but that are not currently the focus of other large-scale knockout mouse programs.

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, the company reports.

VelociGene, combined with Regeneron's other platforms, will not only allow the company to generate its next generation of therapeutic drug candidates but also help the NIH build on the information generated by the Human Genome Project and aid drug discovery and other healthcare efforts outside of Regeneron's walls, Yancopoulos says.

"There was huge excitement and enthusiasm over the completion of human genome sequencing projects." he recalls. "People expected that once you had the sequence of all genes, that would really open up the genome to new targets and drug therapies. And it was a great and necessary first step.

"But the problem is that knowing the sequences doesn't help you figure out what every gene does in normal development, much less in a disease state. And that is why we need high-throughput technologies to create knockout mice and provide them on a massive scale to all researchers."