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The importance of early action
Early intervention in this cascade of events, by favoring the clearance of the amyloid
protein prior to its organization into fibrils and thereby maintaining its solubility, could
clearly change disease outcome. This "disease-modifying" preventative and therapeutic
intervention would be an effective
and innovative approach to arresting the development of amyloid-related diseases. This is
where Neurochem's technology enters into action.
Neurochem's core technology platform resides largely in the ability to design and synthesize
small organic molecules that mimic the sulfated GAG molecules. Capitalizing on the key role
of GAGs in the processes of amyloid fibril formation and deposition, Neurochem has designed
and patented small organic molecules (drug candidates) to compete with these sulfated GAGs
preventing the formation of amyloid fibrils, and the binding of the fibrillogenic
amyloid proteins to the cells. In so doing, the therapeutic molecules inhibit the fibrillogenesis
process. The Company refers to these drug candidates as "GAG mimetics".
The Company believes that this approach introduces a new class of drug candidates for many
of the amyloid-related diseases known to this day, including Alzheimer's disease,
AA Amyloidosis, hemorrhagic stroke due to cerebral amyloid angiopathy and others.
The GAG mimetic

Neurochem's GAGs compete with natural GAGs for the same binding sites on the amyloid
proteins, preventing the association of natural GAGs with the protein.

By preventing natural GAGs from binding to the amyloid proteins, the GAG mimetics can
prevent fibril formation.
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