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Delivery of Transthyretin (TTR) across the Blood Brain Barrier as a Treatment for Alzheimer's Disease

Posted May 25 2010 5:00pm

Description of Invention:
The invention describes products and methods of treating Alzheimer's disease. Alzheimer's disease is characterized by the formation of amyloid plaques and tangles in areas of the brain critical for learning and memory. The products are a transthyretin and other blood brain barrier impermeable proteins transformed into blood brain barrier permeable forms by the coupling of an Inter-Cellular Adhesion Molecule-1 (ICAM-1) targeting agent. Transthyretin binds to, and inhibits amyloid protein from forming plaque deposits. Deposition of amyloid is thought to underlie the disease pathology of Alzheimer's. Thus, this invention treats Alzheimer's by inhibiting the formation of amyloid plaques, which normally would result in amyloid plaque formation, inflammation, and neuronal cell death.

  • Therapeutic for Alzheimer's disease
  • Therapeutic for other amyloid-related diseases

Development Status:
Early stage

Juan J Marugan (NHGRI)

Patent Status:
HHS, Reference No. E-268-2009/0
US, Application No. 61/286,205 filed 14 Dec 2009

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The NIH Chemical Genomics Center (NCGC) is open to collaborating in order to further develop this invention. Please contact Dr. Juan Marugan at for more information about collaborative research opportunities.

Central Nervous System
Central Nervous System - Therapeutics

For Additional Information Please Contact:
Steven Standley Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-4074
Fax: 301-402-0220

Ref No: 2117

Updated: 05/2010

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