Targeting endo-lysosomal dysfunction for the treatment of dementia

Lysosomal dysfunction is at the centre of Parkinson’s disease and other neurodegenerative diseases. 

We are developing small-molecule therapeutics targeting ATP13A2 and ATP10B lysosomal transporters. Both targets are critical for maintaining neuronal health, contribute to neurodegeneration and are linked to human diseases. 

ATP13A2 is a causal gene linked to Parkinson's disease encoding a lysosomal transporter of polyamines, which are key for neuroprotection. When impaired, ATP13A2 disturbs lysosomal and mitochondrial health. 

ATP10B is a candidate risk gene for Parkinson's disease. As a lipid transporter, ATP10B works in concert with GBA1, the major genetic risk factor for Parkinson’s disease, to prevent lysosomal glucosylceramide lipid accumulation. 

ATP13A2 and ATP10B represent high-opportunity targets with first-in-class potential. 

The initial clinical PoC indications are genetic subsets of Parkinson’s disease, with the aim to expand into sporadic Parkinson’s disease and other neurodegenerative disorders.