QurAlis’ QR43 Platform™ is dedicated to discovering and developing precision therapies for TDP-43-related diseases. TDP-43 (TAR DNA-binding protein 43) is a nuclear protein that is highly involved in the processing, splicing, and overall regulation of messenger RNA (mRNA). Nearly 5% of all ALS patients suffer from a mutation in the TARDBP gene that encodes the TDP-43 protein. These mutations cause TDP-43 to move out of the nucleus and into the cytoplasm forming aggregates, impeding the production of various proteins. 

Along with experts in the ALS field, our team developed an in-vitro model to study ALS which has pioneered the discovery of multiple genetic targets in sporadic ALS cases. Examples of this technology include neurodegeneration caused by overly stimulated neurons and the accompanied toxicity of protein aggregates outside of the nucleus.