QurAlis is currently advancing three discovery programs, each addressing a specific component of ALS pathology and a defined ALS patient population based on both disease-causing genetic mutation(s) and clinical biomarkers.
Our programs are aimed at creating disease-modifying, clinically meaningful therapies. They are:
Excitotoxicity in the motor system:
Up to half of ALS patients experience motor system hyperexcitability. This means their motor neurons are overactive, creating an excess of toxic matter that cannot be cleared and thus inhibits neuron function. QurAlis is developing a therapeutic candidate that could potentially regulate this hyperexcitability.
Restoration of autophagy pathways:
Many cases of ALS and FTD are connected to mutated proteins which prevent patients’ motor neurons from clearing out waste – as a result, these neurons are not able to function properly. QurAlis is developing a therapeutic to target an enzyme at the center of this process to bring waste clearance pathways back to normal functioning levels.
The majority of ALS patients show a change in location and a toxic buildup of TDP43 proteins in motor neurons. This is causing these neurons to degenerate, a process believed to be a leading driver of the disease in many ALS patients. QurAlis is developing a therapeutic candidate to counteract TDP43 toxicity and protect neurons against degeneration.