AcuraStem receives $7.5 million CIRM grant for ALS drug AS-241 clinical advancement

AcuraStem has been awarded $7.5 million by the California Institute for Regenerative Medicine to advance AS-241, an antisense oligonucleotide targeting TDP-43 pathology in ALS and FTD. The ASO restores UNC13A expression in patient-derived neurons, with TDP-43 pathology observed in approximately 97% of ALS patients.

AcuraStem has announced that the California Institute for Regenerative Medicine has awarded the company $7.5 million in grant funding to support advancement of its lead clinical candidate, AS-241, toward first-in-human testing, according to an official announcement. AS-241 is an antisense oligonucleotide designed to address a core molecular consequence of ALS and FTD driven by dysfunction of the protein TDP-43. Pathological loss of nuclear TDP-43 leads to cryptic splicing of the UNC13A gene, resulting in loss of a protein essential for healthy synaptic communication.

AcuraStem is a biotechnology company advancing treatments for neurodegenerative diseases including ALS and FTD. TDP-43 pathology is observed in approximately 97% of ALS patients regardless of underlying genetic cause, and is also present in large subsets of patients with FTD and other neurodegenerative disorders. Preclinical studies using AcuraStem's iNeuroRx technology platform demonstrated that AS-241 restores normal UNC13A expression and improves synaptic function in ALS patient-derived neurons.

This development matters for the neurodegenerative disease therapeutics sector, where AS-241 is designed to act broadly across the disease population, addressing a key consequence of TDP-43 pathology shared by the overwhelming majority of ALS patients. This represents a major advance over recently approved Qalsody, which works only for the 2% of patients harboring the SOD-1 mutation.

For the biotechnology industry, CIRM funding undergoes one of the most rigorous scientific and translational review processes, reflecting confidence in the science and therapeutic potential of AS-241. In vivo data supporting safety and durable central nervous system exposure formed the basis for CIRM's decision, with the award essential for moving AS-241 rapidly toward a Phase 1 clinical trial.