Rethinking Transformative ALS Targets, Seek Translational Biomarkers & Propel More Clinical Approvals

The ALS research landscape is evolving rapidly. Insitro’s milestone achievements with Bristol Myers Squibb underscore the potential of computational approaches in target discovery, while Dewpoint Therapeutics’ collaboration with Mitsubishi Tanabe Pharma America to advance TDP-43 condensate modulators highlights the growing interest in innovative molecular mechanisms. At the same time, advancements like QurAlis’ clinical trials for QRL-101 and QRL-201 and breakthroughs in biomarker platforms are reshaping the trajectory of therapeutics in ALS and FTD. 

150+ scientific leaders united in Boston in May to collectively address the most pressing challenges in ALS therapy development—target validation, biomarker utilization, and clinical trial optimization—to accelerate the delivery of effective therapies for ALS and FTD.