AcuraStem Secures $7 Million in Funding Support from NIH and DOD to Accelerate Therapies for ALS and FTD
Summary
AcuraStem Leverages iNeuroRx\xae Platform to Drive Innovative SYF2 and UNC13A Treatments Forward Toward Clinical TrialsPASADENA, Calif., Jan. 30, 2024 /PRNewswire/ -- 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. 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. Opinions, interpretations, conclusions and recommendations are those of the author and are not necessarily endorsed by the Department of Defense.About AcuraStem is a patient-based biotechnology company pioneering treatments for neurodegenerative diseases including sporadic ALS and FTD.