AcuraStem, a biotechnology company developing disease-modifying therapies for amyotrophic lateral sclerosis (ALS), announced on Tuesday that it has received a two-year research grant from non-profit organisation 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 company says that 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.

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 will 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 aims to 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.