PASADENA, Calif., March 10, 2026 /PRNewswire/ — AcuraStem, a patient-based biotechnology company developing disease-modifying therapies for amyotrophic lateral sclerosis (ALS), today announced it has received a two-year research grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function. TDP-43 dysfunction is a central biological hallmark of ALS.
The Target ALS Drug Discovery Consortium award will support mechanistic and translational studies of SYF2, a pre-mRNA splicing factor whose modulation has been shown in preclinical models to restore normal TDP-43 activity and protect motor neurons. Because TDP-43 dysfunction is present in the vast majority of ALS cases, including sporadic disease, targeting SYF2 represents a promising approach with the potential to benefit a broad patient population in ALS.
“For most people living with ALS, the disease is driven by the dysfunction of TDP–43, but there are still no effective treatments that target this core pathology,” said Sam Alworth, M.S., MBA, co–founder and CEO of AcuraStem. “This project will accelerate our understanding of SYF2 biology and help lay the groundwork for future SYF2-targeted medicines.”
Under the award, AcuraStem will collaborate with leading academic investigators, including Philip C. Wong, Ph.D. (Johns Hopkins University), and Wilfried Rossoll, Ph.D. (Mayo Clinic Jacksonville). These collaborations unite complementary strengths in TDP-43–dependent RNA splicing and fluid biomarker development (Wong lab) with cutting-edge proteomics and neuroproteostasis expertise focused on TDP-43 proteinopathy (Rossoll lab), alongside AcuraStem’s patient-derived ALS motor neuron platforms and SYF2-targeted antisense oligonucleotide. Together, this multidisciplinary team will define how SYF2 modulation reshapes TDP-43 RNA processing, protein interactomes, and ALS-relevant neuronal phenotypes, establishing a rigorous mechanistic framework to advance SYF2-targeted therapeutics toward the clinic.
SYF2 was initially identified as a regulator of TDP-43 biology through collaborative research with Justin Ichida, Ph.D. (University of Southern California). Subsequent work by AcuraStem and Ichida lab scientists demonstrated that suppressing SYF2 alleviates TDP-43 pathology and neurodegeneration in ALS models, providing the scientific rationale for advancing SYF2-targeted therapeutics.
About AcuraStem
AcuraStem is a patient-based biotechnology company pioneering treatments for neurodegenerative diseases, including sporadic ALS and frontotemporal dementia (FTD). AcuraStem’s iNeuroRx® platform enables the discovery and development of broadly acting, disease-modifying therapies using patient-derived cellular models.
Contacts:
Kissy Black
Director of Communications, AcuraStem
kblack@acurastem.com
615.310.1894
Roxan Olivas
Lotos Nile
roxan@lotosnile.com
520-954-1634
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SOURCE AcuraStem – Patient-Based Therapeutics
