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Structurally targeted antibodies to aggregation-prone transthyretin

Targeting transthyretin amyloidosis

Transthyretin (TTR) is a protein found in blood that is responsible for transporting important molecules throughout the body.  Normally TTR, which is composed of four identical subunits, is a very stable protein.  However, these subunits occasionally come apart, allowing the single-subunit to partially unravel and expose adhesive amyloid-forming segments, and aggregate with other subunits.  In rare cases hereditary mutations destabilize the TTR structure dramatically promoting amyloid formation and disease.

 

TTR amyloidosis is a rare and progressive disease that is ultimately fatal.  It can manifest in many forms depending on the location of the toxic amyloid deposits in the body. Misfolding of TTR leading to amyloid formation is often caused by hereditary mutations in the protein, however it can also occur in people with a normal TTR protein (called wild-type). In TTR polyneuropathy deposits are found in peripheral nerves.  In TTR cardiomyopathy deposits form within the heart and damage the muscle.

Utilizing a unique screening approach ADRx has developed novel antibodies that sequester aggregation-prone TTR as a strategy to prevent disease.  This antibody is currently in preclinical testing and will quickly progress into the clinic as a companion drug to recently approved treatments.