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- AcuraStem has been awarded a two-year research grant from Target ALS to advance therapeutics targeting SYF2, a pre-mRNA splicing factor that regulates TDP-43 function in amyotrophic lateral sclerosis. - The grant will support mechanistic studies of SYF2 modulation, which has shown promise in preclinical models for restoring normal TDP-43 activity and protecting motor neurons from degeneration. - The research collaboration includes Johns Hopkins University and Mayo Clinic Jacksonville investigators, focusing on TDP-43 dysfunction present in the vast majority of ALS cases. - SYF2 suppression has demonstrated the ability to alleviate TDP-43 pathology and neurodegeneration in ALS models, providing scientific rationale for therapeutic development.
- The ALS Association has awarded $2 million through its Hoffman ALS Clinical Trial Awards Program to support two phase 1 trials testing novel ALS therapies. - Mayo Clinic Jacksonville will test PAS-004, a MEK enzyme inhibitor targeting inflammation and nerve damage, in 12 ALS patients in partnership with Pasithea Therapeutics Corp. - Molefy Pharma SL will conduct first-in-human testing of AP-2, which targets TDP-43 protein imbalances found in 97% of ALS patients. - These early-stage trials aim to generate critical safety, dosing, and biomarker data to accelerate development of effective ALS treatments.
