QurAlis is specifically focused on discovering and developing new therapies for amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease. ALS is a devastating disease which causes rapid cell death in motor neurons, leading to paralysis and an inability to speak or breath, with an average life expectancy of only 3 years. Recent advances in DNA sequencing of patients show that ALS can be caused by mutations in over 25 different genes and is actually a combination of multiple different sub-forms of the disease. It is therefore unlikely that all ALS patients can be treated the same way.
The QurAlis strategy is to go for ALS one gene at a time. We do this by using a transformative system in which cells from ALS patients are used to model the disease in the lab to identify new drugs. Two of the founders of QurAlis, Harvard professors Kevin Eggan and Clifford Woolf have pioneered this technology for ALS, resulting in the discovery of a potential new ALS drug, Ezogabine, which is now tested in a phase 2 clinical trial.
QurAlis is a Cambridge based subsidiary company of Q-State Biosciences, a stem cell biotechnology company that has developed proprietary technologies for which QurAlis has the exclusive rights for use in ALS drug discovery. QurAlis is also licensing drug discovery technologies developed at Boston Children’s Hospital by co-founder and CEO, Dr. Kasper Roet and from Harvard University.
The founders, together with ALS clinicians, other world leaders in ALS research and experienced industry specialists, have developed three discovery programs for QurAlis: (1) Altered motor system excitability (2) Repeat expansion toxicity and (3) Immune system malfunction. Each program has a defined ALS patient population based on the disease-causing mutation(s) and clinical biomarkers, strives for the development of life saving therapy and can work for a larger patient population.
Focusing on specific patient groups defined by genetic mutation and using stem cell technology to build models for each specific group will give QurAlis a high probability of success. If QurAlis can discover and bring new medicines for this devastating disease, the societal and economic impact would be highly significant, with incalculable impact on individual lives and families.