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AcuraStem

Ownership: Private
Development stage: Preclinical

Overview

Platform: iNeuroRx® combines patient-derived stem cell models with AI/machine learning to identify novel targets and therapies across neurodegenerative diseases[3][7]. Pipeline Focus: Multiple preclinical ASOs targeting PIKFYVE/SYF2/UNC13A pathways show promise in both genetic and sporadic ALS/FTD models[5][8]. Recent Milestones: • $580M+ partnership with Takeda for PIKFYVE inhibitors (including lead candidate AS-202)[9] • $7M NIH/DOD funding secured in Jan.2024 to advance SYF2/UNC13A programs toward clinical trials[9]

Therapeutic areas: Neurology
Modalities: Antisense oligonucleotides (ASOs)Small molecules
Industry: Biotech

Drug pipeline

5 assets

AS-202

Preclinical

4 more assets in the full pipeline.

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Partnerships

Takeda PharmaceuticalsCMT Research Foundation

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