27 may 2026
Twenty years ago, we made a bold, early investment in antisense technology, something that had never been tried for neurodegenerative diseases before. That investment led to Qalsody® (tofersen), the first FDA-approved genetically targeted treatment for ALS.
For some people living with a familial form of the disease, called SOD1-ALS, Qalsody has not only slowed progression but shown evidence of restoring some function. It was a watershed moment: proof that ALS can be stopped by targeting its biological roots.
But only about 2% of people living with ALS have a mutation in the SOD1 gene. The challenge now is to expand this progress to the other 98%.
With new data recently reported by QurAlis about its investigational therapy QRL-201, we have a hint at what might be possible.
Building on the Promise of Antisense Technology for Sporadic ALS
QRL-201 is an antisense oligonucleotide (ASO) designed to restore levels of an important protein called stathmin-2 (STMN2), which is essential for nerve cells to build, maintain, and repair their connections.
In most people with ALS, including both sporadic and genetic forms of the disease, stathmin-2 levels are reduced because a protein called TDP-43 stops doing its job properly. Without the help of TDP-43, the instructions for making stathmin-2 get copied incorrectly and too little functional protein is made. This happens with a number of other proteins as well, but across many different studies, stathmin-2 has been shown to be the most downregulated protein due to TDP-43 dysfunction.
According to interim results from a phase 1/2 trial with 69 participants, QRL-201 successfully increased stathmin-2 levels and showed an “encouraging trend” in slowing progression in participants with sporadic ALS. Because this was an early-stage trial designed to assess safety and tolerability, these results are preliminary. Treatment efficacy will need to be assessed in a larger phase 3 trial.
“These results from the ANQUR clinical trial are well aligned with our understanding of STMN2 biology and the potential of STMN2 restoration to have a positive impact on ALS disease progression,” Dr. Kasper Roet, CEO and co-founder of QurAlis, said. “We look forward to advancing the QRL-201 clinical program to deliver a much-needed precision medicine option for people living with ALS.”
Dr. Roet, who was a 2015 Milton Safenowitz Postdoctoral Fellow, has long believed that genetics and precision medicine could fundamentally change the trajectory of ALS therapy.
“It was really the genetics that gave me the belief that ALS is a treatable disease,” he said. “And I believe that even more strongly now.”
Creating a Continuum of Innovation
QRL-201 did not emerge overnight. Its development reflects years of investment across the ALS research ecosystem, including funding from our Lawrence and Isabel Barnett Drug Development Program, which helped support preclinical testing and advance the therapy toward clinical trials.
There is still much more to be done, including a phase 3 trial slated to begin next year, but the significance of these results extends far beyond the potential of a single investigational drug. They reflect the progress that can be made through sustained investment in technology, scientific talent, and drug development.
Belief in the power of antisense technology became an innovation that’s changing what’s possible to achieve with ALS treatment.
A postdoctoral fellowship became a biotech company.
An ASO in the lab became a possible new treatment being tested in clinical trials.
“This is how we are making ALS livable,” said Senior Vice President of Research Dr. Kuldip Dave. “By investing in the cutting-edge technology, emerging talent, and translational research that can improve the lives of people living with ALS today, while bringing us closer to a cure tomorrow.”
Source: ALS Association