Angela Genge, MD, FRCPC, eMBA, on Targeting STMN2 to Rescue Neurons in ALS

“QRL-201, by restoring STATHMIN-2, could stabilize the loss of motor neurons entirely, by returning to normal levels, a protein that we believe is fundamental to the loss of motor neurons when it is knocked down or diminished. We believe that if this is as effective as we see in the cell models, we can actually cause a stabilization of this disease as opposed to the current approved therapies which give functional preservation.”

QuarAlis’ QRL-201 is an antisense oligonucleotide therapy being evaluated for the treatment of amyotrophic lateral sclerosis (ALS) in a first-in-human, phase 1 study (NCT05633459). It is designed to restore STATHMIN-2 (STMN2) and the trial is evaluating 8 dose cohorts of 8 patients each. Angela Genge, MD, FRCPC, eMBA, clinical director, executive director, clinical research unit, Montreal Neurological Institute, and chief medical officer, QurAlis, presented on the therapy at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas.

CGTLive spoke with Genge to learn more about the advantages of QRL-201 and how its mechanism of restoring STMN2 may rescue the disease phenotype seen in ALS. She discussed the need for disease-modifying therapies in ALS compared to the current treatment landscape of symptom-relieving therapies. She also touched on unmet needs in ALS and research that remains to be done to better treat the disease.

Read more coverage of the 2023 MDA Conference here.