CIRM Awards AcuraStem $7.5 Million in Grant Funding to Advance AS-241 Toward a First-in-Human Trial for ALS and FTD

PASADENA, Calif., April 28, 2026 /PRNewswire/ -- AcuraStem, a biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company $7.5 million in grant funding to support the advancement of its lead clinical candidate, AS-241, toward first–in–human testing.

AS-241 is an antisense oligonucleotide (ASO) 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 that is essential for healthy synaptic communication. TDP-43 pathology is observed in approximately 97% of ALS patients, regardless of the underlying genetic cause, and is also present in large subsets of patients with FTD and other neurodegenerative disorders.

By addressing a key consequence of TDP–43 pathology that is shared by the overwhelming majority of ALS patients, AS-241 is designed to act broadly across the disease population. This represents a major advance over the recently approved drug Qalsody®, which, while effective and disease-modifying, only works for the 2% of patients who harbor the SOD-1 mutation. 

Preclinical studies using AcuraStem's iNeuroRx® technology platform have demonstrated that AS-241 restores normal UNC13A expression and improves synaptic function in ALS patient-derived neurons. These findings, along with in vivo data supporting safety and durable central nervous system exposure, formed the basis for CIRM's decision to fund the program.

"CIRM funding undergoes one of the most rigorous scientific and translational review processes in the field," said Sam Alworth, M.S., MBA, co–founder and CEO of AcuraStem. "Their support reflects strong confidence in the quality of our science and the therapeutic potential of AS-241. This award is essential for helping us move AS-241 rapidly toward a Phase 1 clinical trial, and we are honored by CIRM's endorsement of this program." 

About AcuraStem 

AcuraStem is a patient–based biotechnology company pioneering broadly acting treatments for neurodegenerative diseases including sporadic ALS and FTD.

AcuraStem's iNeuroRx® platform enables the discovery and development of disease–modifying therapies based on human patient biology. AcuraStem's research is supported by grants and partnerships from leading public and private organizations focused on accelerating transformative therapies for patients.

For additional information, please visit acurastem.com and follow AcuraStem on LinkedIn, Facebook, and X.

About the California Institute for Regenerative Medicine (CIRM)

The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state's biotechnology economy, and expand access to transformative treatments. Today, CIRM is pioneering new models of therapy development and accelerating medical breakthroughs that change lives — in California and around the world. For more information, visit www.cirm.ca.gov.

Contacts:

Kissy Black
Director of Communications, AcuraStem
[email protected]
615.310.1894

Roxan Olivas
Lotos Nile
[email protected]
520-954-1634

SOURCE AcuraStem - Patient-Based Therapeutics