AcuraStem has received USD 7.5 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to advance its AS-241 ALS treatment candidate toward a first-in-human trial, the Pasadena, California-based company announced on April 28, 2026.
AS-241 is an antisense oligonucleotide (ASO) designed to counter a molecular consequence of TDP-43 protein dysfunction, a pathological feature observed in approximately 97% of ALS patients regardless of underlying genetic cause, and in substantial portions of patients with frontotemporal dementia. When TDP-43 function is lost in the nucleus, the UNC13A gene undergoes aberrant cryptic splicing, resulting in loss of a protein required for normal synaptic communication. AS-241 is designed to suppress that cryptic splicing and restore UNC13A expression. Preclinical studies conducted using AcuraStem's iNeuroRx platform in ALS patient-derived neurons, alongside in vivo data characterizing safety and central nervous system exposure, formed the basis for the CIRM award.
AcuraStem positions AS-241 as a potential broad-population therapy, in contrast to existing approved options. The company noted that Qalsody (tofersen), a recently approved ASO for ALS, addresses only the approximately 2% of patients who carry a SOD1 mutation. Because TDP-43 pathology is present across the large majority of ALS cases and extends into FTD and other neurodegenerative conditions, a therapy targeting a downstream consequence of that pathology could, if clinical data support the preclinical findings, be relevant to a wider patient group. That framing reflects a design rationale rather than a demonstrated clinical outcome, as AS-241 has not yet entered human testing.
The award reflects CIRM's role as a California state agency established to accelerate stem cell and gene therapies toward patients with unmet medical needs. Since its creation in 2004 following a voter-approved mandate, CIRM has deployed USD 8.5 billion across a portfolio spanning clinical trials, workforce development, and biotechnology infrastructure. The agency's review process for awards of this type evaluates both scientific quality and translational readiness, and the AS-241 program was presented as meeting those criteria on the basis of its preclinical efficacy, safety, and durable CNS exposure data.