Under the terms of the contract, Aptagen will apply its proprietary technology to develop aptabodies™ -- synthetic "molecular bullets" designed to deliver a therapeutic payload to diseased cells.
Aptagen's technology will be used as a research tool to expedite drug development in a leading mouse model of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. ALS is a progressive and fatal neurodegenerative disease with no known cause or cure.
The integration of aptabodies into the Institute's arsenal of technology is an integral part of its scientific plan announced in January. Through its collaboration with the Muscular Dystrophy Association's Augie's Quest ALS initiative, the Institute will apply the aptabody technology in investigating the potential of gene therapy for treating ALS.
"The use of aptabody conjugates to target a therapeutic to a specific cell type is a powerful tool that removes some of the limitations of drug delivery that utilize engineered viruses," said Steven Perrin, Ph.D., chief scientific officer at ALS TDI.
"Aptabody drug delivery may reduce some of the inefficient trial and error of developing small molecules that must cross the blood brain barrier to reach their respective targets in the central nervous system," Perrin added.
"Traditional drug discovery strategies against devastating diseases like ALS have been slow to reach the clinic," said Tom Caltagirone, Ph.D., president and CEO of Aptagen. "We are optimistic about ALS TDI's initiative to aggressively explore aptibody technology as a viable solution for rapidly developing successful drug candidates against ALS."