Research aimed at developing “molecular bullets” for ALS drug delivery
CAMBRIDGE, Mass., Jan. 7 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI) today announced it has signed a research and development agreement with Aptagen LLC, of Jacobus, Penn. Under the terms of the contract, Aptagen will apply its proprietary technology to develop aptabodies(TM) -- 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.”
Aptabodies can be exploited in preclinical therapeutic development, either as a research tool or potentially as a novel therapeutic delivery system for treating disease. An aptabody is comprised of a piece of RNA that acts as a molecular scaffold for attaching drug molecules. Like antibodies, aptabodies can bind to cell targets with high affinity and specificity. Because development of aptabody-based drugs does not require prior knowledge of a disease target, the technology can dramatically reduce the time and cost of drug development.
“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.”
About ALS Therapy Development Institute
The ALS Therapy Development Institute (http://www.als.net), based in Cambridge, Mass., operates the world’s largest research and development program focused exclusively on ALS. Its staff of 30 scientists and research technicians work on behalf of ALS patients to discover and advance novel therapeutics for treating and ultimately curing ALS. The non-profit biotechnology institute excels in identifying novel disease targets, discovering compounds that may act against these targets, and screening potential treatments for clinical development.
About Augie’s Quest
Fitness pioneer Augie Nieto started Augie’s Quest (http://www.augiesquest.org) in conjunction with MDA’s ALS Division. Nieto is co-founder and former president of Life Fitness of Chicago, and chairman of Octane Fitness. He and his wife, Lynne, serve as co-chairpersons of MDA’s ALS Division. Nieto received a diagnosis of ALS in March 2005.
About MDA
MDA (http://www.mda.org) is the world’s largest provider of ALS services and funder of ALS research. Over the years, it has expended more than $200 million in this effort. It operates 225 neuromuscular disease clinics across the country and 37 ALS-specific research and care centers. In 2007, MDA entered into a $36 million collaboration with ALS TDI to develop drugs for ALS.
About Aptagen
Aptagen, based in Jacobus, Penn., is an early-stage biopharmaceutical company developing a proprietary technology for creating libraries of novel molecules called aptabodies that can be used either as a research tool or potentially as a novel therapeutic delivery system for treating disease.
CONTACT: Robert Goldstein of ALS TDI, +1-617-441-7295, rgoldstein@als.net
Web site: http://www.als.net/
http://www.augiesquest.org//
http://www.mda.org/
