03/10/2026
🚨 Latest News:
AcuraStem has been awarded a two-year Drug Discovery Consortium grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function.
TDP-43 dysfunction is present in approximately 97% of ALS cases, including sporadic disease, and remains without an approved therapy that directly addresses it. In preclinical ALS models, suppressing SYF2 restores normal TDP-43 activity and protects motor neurons from degeneration, representing a potential therapeutic path for the broad ALS patient population.
The grant will fund mechanistic and translational studies in collaboration with:
Philip C. Wong, Ph.D., Johns Hopkins University, specializing in TDP-43 RNA splicing and fluid biomarker development Wilfried Rossoll, Ph.D., Mayo Clinic Jacksonville Inc., specializing in proteomics and neuroproteostasis in TDP-43 proteinopathy
Their expertise, combined with AcuraStem's iNeuroRx® patient-derived motor neuron platforms and SYF2-targeted antisense oligonucleotide program, will focus on defining how SYF2 modulation reshapes TDP-43 biology in ALS neurons and establishing the mechanistic foundation for clinical development.
Every experiment, every late night reviewing data, every iteration on the science that got us to this point was carried by a team that believed this work could make a difference for ALS patients. This grant exists because of them:
Philip Wong
Wilfried Rossoll
Justin Ichida
Marcel van der Brug
Wen-Hsuan (Wen) Chang
Zhihua Feng
Shu-Ting (Michelle) Hung
PhD Emily Lee
Thank you to Target ALS for your continued investment in ALS drug discovery.
Full press release at: https://pxllnk.co/TargetALSGrant
