California, USA-based biotech AcuraStem has entered into a license agreement with Japan’s largest pharma company Takeda (TYO: 4502) to develop and commercialize AcuraStem's PIKFYVE targeted therapeutics including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of amyotrophic lateral sclerosis (ALS).
PIKFYVE is a novel therapeutic target for ALS and may also be relevant for treating additional TDP-43 proteinopathies such as frontotemporal dementia (FTD). AcuraStem's therapeutic strategy for PIKFYVE focuses on addressing neurodegeneration by expelling toxic protein aggregates and protecting healthy neuronal function.
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