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
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.
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.
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.
SOURCE ALS Therapy Development Foundation