An RNA-based approach to prion disease

There’s an interesting new entrant into Ionis Pharmaceuticals’ preclinical pipeline: A drug candidate for prion diseases. This a family of rare neurodegenerative diseases caused by prions, which are misfolded proteins that can transmit their abnormal structure to healthy proteins — particularly in the brain. The most famous illness triggered by prions is “mad cow disease,” but there are genetic versions of these disorders as well. Some forms lead to dementia, disability, and then death.

A husband-and-wife team began tackling prion-based illnesses with a genetic underpinning after the wife, Sonia Vallabh, learned she carried a mutation leading to the disease. Now their work seems to be coming to fruition — with an antisense oligonucleotide drug candidate in the works at Ionis.