Business:
ALS cure - Small Molecules, ASO
Drug notes:
QRL-201/QRL-203 Clin0 FTD, Clin0 PLS; QRL-101 Clin1 ALS, Clin0 FTD; QRL-204 Clin0 ALS/FTD, Clin0 FTD
About:
QurAlis is using advances in precision medicine to develop therapeutics for neurodegenerative diseases. Amyotrophic lateral sclerosis (ALS), is a progressive neurodegenerative disease that reduces muscle function and control. By examining the disease-causing genetic alterations linked with ALS, QurAlis is targeting these disease mechanisms using human neuronal stem cell models from ALS patients - this includes the gene that encodes the protein TDP-43. QuAlis has a second platform, FlexASO, a proprietary anti-sense oligonucleotide splice modulator platform to selectively modulate RNA levels to change the expression of genes connected to disease. With these approaches, QurAlis has already established a pipeline of programs in clinical stages.
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