New Compounds For The Treatment Of Diseases Related To Protein Misfolding

Tech ID: 27261 / UC Case 2012-615-0


UCLA researchers in the Department of Neurology with an international team of scientists have developed compounds for therapeutic use in protein misfolding diseases.


Protein misfolding is the underlying cause of a number of neurodegenerative diseases such as Alzheimer’s disease (AD) and Parkinson’s disease (PD) as well systemic diseases such as Type-2 diabetes and prion diseases. In each of these diseases, a protein adopts an altered conformation composed of β-sheets and is found in large deposits that are toxic to cells. For example, AD patients are known to have large β-sheet rich deposits composed of the protein, amyloid-β. Currently, these diseases have no therapeutics and with a global aging population present a huge financial burden.


UCLA researchers have developed novel compounds based on the β-sheet rich structure adopted by the proteins in the aggregated state. The small molecules bind the β-sheet structure and suppress their formation. They also disassemble pre-formed aggregates robustly. The small molecules have shown to be neuro-protective against amyloid-β induced aggregation and toxicity in cultured cells.


  • Therapeutic in Alzheimer’s disease
  • Therapeutic in Parkinson’s disease
  • Therapeutic for protein misfolding disease


  • Applicable to broad range of protein misfolding diseases
  • Prevents formation of new aggregates
  • Breaks pre-formed aggregates

Related Materials

Patent Status

Country Type Number Dated Case
United States Of America Issued Patent 8,481,494 07/09/2013 2012-615
European Patent Office Published Application EP2493859 09/05/2012 2012-615


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  • Bitan, Gal

Other Information


Protein misfolding, amyloid, therapeutics, amyloid-ß, Alzheimer’s disease, Parkinson’s disease, Type-2 diabetes

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