Knockout model to be developed for Rare Disease Science Challenge winner
Hudson, New York - July 29, 2013 - Taconic, a leading provider of life sciences solutions, announced it has begun a custom model generation project for Jonah's Just Begun-Foundation to Cure Sanfilippo, winner of a national science challenge for which Taconic served as a sponsor. Taconic is donating a custom-generated knockout model to the organization, demonstrating its strong commitment to advancing rare disease research.
The Jonah's Just Begun project was chosen as a winner in the Rare Disease Science Challenge, which is hosted by Assay Depot and Rare Genomics Institute to provide donated services to accelerate rare disease research. Jonah's Just Begun won the Taconic-sponsored Platinum Award of a Custom Mouse Model from among 77 applicants in its category.
"Most rare diseases have a genetic component, and studying the effects of genomic alterations in vivo is a critical first step in understanding how they develop," said Todd Little, President and CEO, Taconic." By generating a custom knockout model for Jonah's Just Begun, Taconic is supporting Sanfilippo Syndrome research with an essential tool to improve understanding of the disease's mechanism, an indispensable precursor to therapy development." Taconic is the industry's most experienced custom mouse model generator, with in-depth expertise in mutations that model human physiology and disease.
Sanfilippo Syndrome is a genetic metabolism disorder that prohibits the proper breakdown of the body's sugar molecules, one of a group of diseases called mucopolysaccharidoses (MPS). The debilitating disease first affects the central nervous system, causing severe brain damage, and typically results in hearing loss, vision loss, organ damage, and bone deformities.
"Mouse models are so critical to the development of new therapies, that it is difficult to imagine any significant progress can be made without them," said Patricia Dickson, MD, principal investigator, medical genetics, LA Biomed at Harbor-UCLA Medical Center. "An MPS IIID mouse model will greatly accelerate proof-of-concept studies and new innovations into treatments for kids and adults suffering from Sanfilippo D syndrome."
Each year, only about four children are born in the U.S. with Sanfilippo Syndrome Type D (the type targeted by this model) and those patients live to age 25 on average - a clear indication that while the disease is rare, patients' lives can be improved and extended through the right therapy. An estimated 1,600 Americans have one of the four types of Sanfillipo Syndrome, and all can potentially benefit from new therapies developed with the help of the Taconic custom model.
Jill Wood, founder and CEO of Jonah's Just Begun, sees great value in Taconic's custom model. "There are few federal dollars devoted to rare disease research and fewer still for those with the smallest patient populations," said Wood, whose 4-year-old son Jonah was diagnosed with Sanfilippo Syndrome type C at 22 months. "Taconic's custom model will provide a research tool that is sorely needed in our quest to understand this disease and develop effective treatments."
Taconic is a global provider of genetically engineered mouse and rat models and services. As a full-service industry leader, founded in 1952, Taconic helps clients acquire, test, develop, breed, cryopreserve, prepare, and distribute highly relevant research lines worldwide. Headquartered in New York's Hudson River Valley, Taconic operates six breeding facilities and three service laboratories in the U.S. and Europe and maintains over 850 employees committed to technological innovation. Taconic's products and services are used by over 1,250 companies and academic research institutions in nearly 50 nations worldwide.