AcuraStem Secures $7 Million in Funding Support from NIH and DOD to Accelerate Therapies for ALS and FTD :: Acurastem
Summary
AcuraStem Leverages iNeuroRx\xae Platform to Drive Innovative SYF2 and UNC13A Treatments Forward Toward Clinical TrialsPASADENA, 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 achievementbuilding upon the recent licensing agreement with Takeda worth $580 million to develop and commercialize AcuraStems PIKFYVE targeted therapeutics including AS-202enables the company to advance multiple programs towards clinical trials. 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. 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 UNC13As 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 Ichidas lab at USC, AcuraStems extensive research on patient neurons via the iNeuroRx\xae 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).AcuraStems 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-43s absence from the nucleus.