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CSM-101

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Preclinical TRPML1 agonist designed to restore lysosomal function in genetically driven Parkinson's-related disorders.

Modality
oral CNS-penetrant small-molecule TRPML1 agonist
Availability (US)
Late-stage preclinical and IND-enabling development as of March 10, 2026; no human trial or approval established
Biomarkers
TRPML1 activation; lysosomal function; toxic lipids; alpha-synuclein; neuroinflammation
Hallmark targeted
disabled autophagy, loss of proteostasis, mitochondrial dysfunction, chronic inflammation
Kind
investigational drug