ALS Therapy Development Institute Signs Aptabody(TM) Discovery Agreement With Aptagen

Research aimed at developing "molecular bullets" for ALS drug delivery

Jan 07, 2008, 00:00 ET from ALS Therapy Development Foundation

    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 (, 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
 ( 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 ( 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.

SOURCE ALS Therapy Development Foundation