SAN DIEGO., December 07, 2025. AcuraStem presented new poster data at the 36th International Symposium on ALS/MND, held December 5–7, 2025 in San Diego, USA, at the Loews Coronado Bay Resort. The Symposium, organized by the MND Association, is the largest annual conference dedicated to ALS and MND research and brings together researchers from around the world to share new findings.
At the Symposium, AcuraStem presented two posters reporting data generated in iNeuroRX®, the company’s gold-standard platform that builds patient-derived human neurons to model ALS and FTD biology and test ASO candidates.
In TST-08, “Assessment of an UNC13A cryptic exon skipping antisense oligonucleotide on restoring neuronal activity in iPSC-derived neuron models,” AcuraStem evaluated an UNC13A cryptic exon skipping ASO in a TDP-43 depletion model. The poster describes how loss of nuclear TDP-43 can drive UNC13A cryptic exon inclusion, reducing UNC13A RNA and protein levels and impairing synaptic function. Using patch-clamp electrophysiology and an intensity-based glutamate sensing assay (iGluSnFR), the team reported reduced neuronal activity after TDP-43 depletion and a significant rescue of activity deficits following UNC13A ASO treatment.
AcuraStem also presented TST-10, “Assessment of SYF2 antisense oligonucleotides in an exploratory toxicity study,” focused on selecting SYF2 ASO candidates based on in vivo tolerability. In an eight-week study following intracerebroventricular administration in adult mice, the team screened more than 25 ASO candidates and tracked both early and delayed safety signals using in-life observations, histopathology, markers of glial activation, and motor testing. The poster reported that some candidates appeared tolerable early but later showed adverse findings, and that this profiling approach identified the most well-tolerated SYF2 ASO candidates for further development.
For registered virtual delegates, Symposium content remains available on demand until March 4, 2026 at symposium.mndassociation.org
For more information about AcuraStem’s work or to explore collaboration opportunities, contact info@acurastem.com.