AcuraStem Leverages iNeuroRx® Platform to Drive Innovative SYF2 and UNC13A Treatments Forward Toward Clinical Trials

PASADENA, Calif., January 30, 2024 — AcuraStem, a patient-based biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, announced today that it has successfully raised nearly $7 million in grant funding for ALS / FTD research from the National Institutes of Health (NIH) and the Department of Defense (DOD).

This latest achievement—building upon the recent licensing agreement with Takeda worth $580 million to develop and commercialize AcuraStem’s PIKFYVE targeted therapeutics including AS-202—enables the company to advance multiple programs towards clinical trials.

"We are thrilled to receive continued support from NIH and the DOD,” said Sam Alworth, MS, MBA, AcuraStem co-founder and CEO. “Leaders from the scientific community selected AcuraStem for these awards through a rigorous peer-review process, which demonstrates the excellence of our research."

AcuraStem has made a significant advancement in understanding the pathophysiology of ALS, focusing on the crucial role of UNC13A. An overwhelming majority of ALS cases, and roughly half of FTD cases, are characterized by TDP-43 pathology. TDP-43, a RNA-binding protein typically found in the nucleus, is mislocalized to the cytoplasm in affected neurons, leading to RNA processing errors. These errors result in the incorporation of a cryptic exon into the UNC13A messenger RNA, causing a loss of UNC13A protein. This loss is further exacerbated by a risk variant in the UNC13A gene, closely linked to the TDP-43 binding site and associated with reduced survival in ALS and FTD patients. AcuraStem's iNeuroRx® platform has successfully replicated UNC13A pathology in patient neurons, enabling the rapid development and testing of antisense oligonucleotides (ASOs). These ASOs effectively suppress the cryptic exon and restore normal UNC13A function, offering a promising therapeutic approach for TDP-43 proteinopathies and a deeper understanding of UNC13A's role in disease progression.

Additionally, AcuraStem has made a pivotal discovery with its focus on SYF2, a novel target in TDP-43 pathology. In collaboration with co-founder Dr. Justin Ichida’s lab at USC, AcuraStem's extensive research on patient neurons via the iNeuroRx® platform, coupled with a comprehensive bioinformatic analysis, identified SYF2 as a key therapeutic target. SYF2, a pre-mRNA splicing factor, plays a crucial role in the regulation of splicing affected by the depletion of nuclear TDP-43, a common feature in most ALS cases (Linares GR et al Cell Stem Cell 2023).

AcuraStem’s findings highlight that SYF2 can address both the toxic aggregation of TDP-43 in cytoplasm and also counter the widespread gene dysregulation, including critical genes like UNC13A and STMN2, caused by TDP-43's absence from the nucleus. Targeting SYF2 could offer a more direct and potentially effective therapeutic strategy compared to addressing each dysregulated gene individually.

Funding from NIH and the DOD was awarded through rigorous peer-reviewed processes, suggesting the award matches the health-related missions established by each agency.

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This project will be supported by an NIH grant (R44AG085411). The content is solely the responsibility of the authors and does not necessarily represent the official views of NIH.

This work was supported by the Office of the Assistant Secretary of Defense for Health Affairs and the Defense Health Agency J9, Research and Development Directorate, or the U.S. Army Medical Research Acquisition Activity at the U.S. Army Medical Research and Development Command, in the amount of $839,898.40 through the ALSRP under Award No. HT9425-23-1-0469. Opinions, interpretations, conclusions and recommendations are those of the author and are not necessarily endorsed by the Department of Defense.




About AcuraStem

AcuraStem is a patient-based biotechnology company pioneering treatments for neurodegenerative diseases including sporadic ALS and FTD. AcuraStem’s best-in-class disease modeling platform, iNeuroRx®, enables the discovery of innovative, effective, and broadly acting treatments. The team’s strong expertise in ASO technology provides for the rapid advancement of treatments to the clinic. AcuraStem’s research is funded in part by support from the Alzheimer’s Drug Discovery Foundation, Harrington Discovery Institute, Alzheimer’s Association, Rainwater Charitable FoundationMuscular Dystrophy AssociationDepartment of Defense and the National Institute of Neurological Disorders and Stroke.


Kissy Black
Director of Communications, AcuraStem