The Spinal Muscular Atrophy Foundation Launches Collaboration for New Disease Models

New York, NY – October 4, 2005 – The Spinal Muscular Atrophy Foundation announced an agreement today with Regeneron Pharmaceuticals, Inc. to develop new animal models of spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers.. The collaboration will exploit Regeneron’s proprietary VelocigeneTM technology to rapidly generate and test new genetic models in murine embryonic cells and to build out the toolkit for SMA therapeutic research. Animals produced from these cells will be used by investigators and drug developers to identify targets and treatments for the disease. VelocigeneTM technology allows for the “humanization” of large portions of the murine genome, allowing for models that may closely approximate human genetic diseases such as SMA.

“This is an extraordinary opportunity to further our understanding of Spinal Muscular Atrophy. We must learn more about genes that influence the course of SMA, and we must develop new therapies based on that knowledge,” said Gerald Fischbach, MD, Dean of the Columbia University Medical Center and a member of the Foundation’s Board of Directors. “Bringing together new technologies and extraordinary scientists is the best way forward.”

The terms of the agreement provide $1.65 million in funding to Regeneron over the collaboration period.

“It is important that we capitalize on these new technologies as quickly as possible,” added SMA Foundation President Loren Eng. “As unbelievable as it may seem, there are no proven treatments for SMA. We are optimistic that breakthroughs in scientific processes like Regeneron’s Velocigene technology will accelerate new therapeutic development.”

About Spinal Muscular Atrophy

Spinal muscular atrophy is a genetic, motor neuron disease characterized by the wasting of skeletal muscles. Caused by progressive degeneration of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy and premature death due to respiratory problems. The SMA Foundation estimates that there are currently over 50,000 people suffering from SMA in the United States, Europe, and Japan and that the market potential for a drug to treat SMA ranges from $500 million to $1 billion dollars annually.

About Velocigene™

Regeneron’s Velocigene™ technology allows for custom and precise manipulation of very large sequences of DNA to produce almost any desired genetic change in murine embryonic stem cells, and further allows for the rapid generation of mice from these embryonic stem cells. Many of the steps in the Velocigene™ process are automated and roboticized. In addition to allowing for rapid generation of conventional gene “knockouts” and “transgenics”, Velocigene™ technology also enables extensive and highly customized genetic manipulations such as those involving exchange of large portions of the mouse genome with the corresponding human genetic sequences, termed “humanizations”. Such humanizations can be very useful in developing models of human genetic diseases. Regeneron scientists have also utilized Velocigene™ technology to generate VelocImmune mice, in which large portions of the mouse immune genome have been humanized, generating mice with humanized immune systems that can rapidly and efficiently generate human antibodies to therapeutic targets of interest.

About the Spinal Muscular Atrophy Foundation

The SMA Foundation is a nonprofit organization founded in 2003 that is dedicated to finding a treatment and potential cure for Spinal Muscular Atrophy (SMA). The Foundation provides funding for the full range of research from basic to clinical work conducted in academic laboratories as well as corporate therapeutics development: since its inception, the Foundation has awarded nearly $20 million in sponsored research agreements. In addition, the Foundation is committed to raising awareness and generating support for increased federal and corporate funding for the disease. For more information on the Spinal Muscular Atrophy Foundation, visit or call (646) 253-7100.

SMA Foundation contacts:

Cynthia Joyce
(646) 253-7100
[email protected]

Bryan deCastro
(631) 495-9177
[email protected]