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SYF2 Program

ALS

DiscoveryActive

Key Facts

Indication

ALS

Phase

Discovery

Status

Active

Company

AcuraStem

About AcuraStem

AcuraStem, founded in 2016 and based in Pasadena, CA, is a private, pre-clinical biotech developing disease-modifying therapies for neurodegenerative diseases like ALS and FTD. Its core innovation is the iNeuroRx® platform, which utilizes patient-derived cellular models to identify and validate novel therapeutic targets, aiming to overcome the high failure rates of traditional animal models. The company has built a pipeline targeting mechanisms like PIKFYVE and SYF2 and is led by a team with expertise in neuroscience, stem cell biology, and drug development. AcuraStem is currently pre-revenue and funded by grants and venture capital.

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Therapeutic Areas

Amyotrophic Lateral Sclerosis (ALS)Frontotemporal Dementia (FTD)Neurodegenerative Diseases

Other ALS Drugs

Drug	Company	Phase	Status
Stem Cell Program	BioArctic	Research	Active
Eolo40 NCE	Eolo Pharma	Discovery	Active
New targets program	Treeway	Preclinical	Active
Target-03	Verge Genomics	Discovery	Active
Target-04	Verge Genomics	Discovery	Active
BIIB105 (ATXN2 ASO)	Biogen	Phase 2	Active
Undisclosed Program	bioarctic-ab	Discovery	Research