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1 filing since 2026 · no dollar figures disclosed
AcuraStem is a Pasadena, California biotechnology company developing antisense oligonucleotide therapeutics for neurodegenerative disease, principally ALS and frontotemporal dementia. Its iNeuroRx platform combines patient-derived neuron disease models with AI and bioinformatics to find targets, and produced the discovery of PIKFYVE as an ALS/FTD target published in Nature Medicine in 2018. Lead programs include PIKFYVE-targeting and UNC13A-targeting candidates, and the company licensed its PIKFYVE program to Takeda in a deal worth up to $580 million.
What sets it apart
Targets are discovered in neurons derived from actual patients rather than animal models, which the company argues is why its candidates aim at sporadic ALS and FTD - the large majority of patients - instead of only the rare familial genetic subsets most ALS programs address.
A drug discovery platform that models neurodegenerative disease in patient-derived neurons and applies AI and bioinformatics to identify and validate therapeutic targets, then develops antisense oligonucleotide drugs against them.
