PASADENA, Calif., April 28, 2026

AcuraStem has received $7.5 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to accelerate development of AS-241, its lead antisense oligonucleotide (ASO) therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The funding will support the advancement of AS-241 toward a first-in-human Phase 1 clinical trial, marking a major milestone for the company’s mission to develop broadly acting treatments for neurodegenerative diseases driven by TDP-43 pathology.

AS-241 Targets the Core Driver of ALS and FTD

AS-241 is designed to address one of the most important biological drivers of both ALS and FTD: dysfunction of the protein TDP-43, which is observed in approximately 97% of ALS patients and in large subsets of FTD patients. Loss of normal nuclear TDP-43 function causes abnormal cryptic splicing of the UNC13A gene, leading to reduced production of a protein essential for healthy synaptic communication and neuronal survival.

By restoring normal UNC13A expression, AS-241 aims to directly target a shared molecular consequence across the majority of ALS patients, regardless of genetic background. This broad applicability makes it significantly different from mutation-specific therapies such as Qalsody®, which is approved only for the small percentage of patients carrying the SOD1 mutation, representing approximately 2% of ALS cases.

Strong Preclinical Data Supports Clinical Advancement

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Preclinical studies conducted using AcuraStem’s proprietary iNeuroRx® platform demonstrated that AS-241 successfully restored normal UNC13A expression and improved synaptic function in ALS patient-derived neurons. Additional in vivo studies also showed strong safety signals and durable central nervous system (CNS) exposure, supporting the therapy’s readiness for clinical development.

These findings formed the scientific basis for CIRM’s decision to fund the program. According to CEO Sam Alworth, the grant reflects strong validation of both the therapeutic potential of AS-241 and the quality of AcuraStem’s translational science. The company plans to use the funding to accelerate IND-enabling work and move rapidly toward initiating its first Phase 1 study.

Expanding Innovation in Neurodegenerative Disease Treatment

AcuraStem is focused on developing disease-modifying therapies for sporadic ALS, FTD, and other neurodegenerative diseases using patient-derived biological models rather than traditional animal-only discovery systems. Its iNeuroRx® platform enables precision therapeutic development based on real human disease biology, improving translational relevance and increasing the probability of clinical success.

CIRM, one of the most significant regenerative medicine funding agencies in the United States, has invested over $8.5 billion since 2004 to accelerate cell and gene therapies for patients with unmet medical needs. Its support for AS-241 highlights the growing urgency and opportunity in developing effective therapies for devastating neurological disorders where treatment options remain limited.

With strong scientific validation, significant non-dilutive funding, and a clear path toward clinical trials, AcuraStem is positioning AS-241 as a potentially transformative therapy for ALS and FTD, offering new hope for patients affected by these progressive and life-threatening neurodegenerative diseases.

Source: AcuraStem press release