Cortico-Basal Degeneration Press release 29Oct13

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NEWS RELEASE:
NsGene, Inc.
225 Chapman Street Suite 201
Providence, RI 02905
NsGene receives grant to develop its Brain Repair Technology for the treatment Cortico-Basal
Degeneration (CBD), a rare neurodegenerative disorder
Karin & Sten Mortstedt CBD Solutions, part of a non-profit foundation, has awarded $545,000 for
NsGene to carry out preclinical development of its Brain Repair therapeutic platform for the treatment
of cortico-basal degeneration (CBD). Professor Peter Davies, PhD, North Shore Hospital, New York, and
Dr. Johan Lundkvist, PhD, at Swedish- based AlzeCure, will collaborate in the research. The funding will
support the investigation of brain delivery of antibodies against a protein called tau which is closely
linked with the pathology of the disease. CBD is a neurodegenerative disorder characterized by atrophy
and nerve cell loss in multiple areas of the brain. Onset of the disease typically occurs around 60 years
of age and symptoms are very similar to those observed in Parkinson’s disease, namely: impaired
balance, muscle rigidity and muscular jerks, halting speech, and cognitive as well as visual-spatial
disturbances. Patients eventually lose the ability to walk.
Recent data has shown that aggregation of the protein Tau plays a central role in the pathology of CBD
by rendering specific brain neurons dysfunctional. A therapeutic that could lower the levels of tau has
the promise to protect the neurons and to halt progression of the disease. NsGene will investigate its
novel Brain Repair technology to deliver therapeutic antibodies directly to the brain, and if successful
will move the technology forward into safety studies and then clinical trials. The clinical Brain Repair
platform is a slender, 1 millimeter in diameter, cylindrical device whose distal end consists of a semipermeable hollow fiber containing a human cell line engineered to produce the therapeutic protein of
choice, and for the CBD indication, the secreted therapeutic will be antibodies against Tau. The clinically
validated device is implanted in the brain and the cells contained within secrete the therapeutic
antibodies that will lower the levels of Tau. This Brain Repair technology potentially solves a significant
technical challenge in treating neurodegenerative disorders, namely the delivery of therapeutic
antibodies to the brain in a safe and efficient manner and in concentrations high enough to see a
positive effect. Currently there are no efficacious treatments for patients struggling with this debilitating
disease.
About Karin & Sten Mortstedt CBD Solutions
CBD Solutions is dedicated to finding a cure for CBD. This is an enormous task and can only be
accomplished through collaboration. CBD Solutions supports and works in partnership with world
leading scientists active within the field of CBD research and are actively seeking new partners
interested in new therapeutic opportunities in CBD (www.cbdsolutions.se).
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