Casma Therapeutics is engineering therapies that target the cell’s autophagy and lysosomal systems to eliminate disease-causing cellular waste. Its lead program, CSM-101, is an orally bioavailable, brain-penetrant small-molecule agonist of the lysosomal ion channel TRPML1 intended to restore lysosomal function and provide disease-modifying benefit in Parkinson’s disease. In preclinical studies, CSM-101 demonstrated high CNS exposure, reduced toxic lipid accumulation, lowered neuroinflammation, decreased alpha-synuclein levels, and preserved dopaminergic neurons in Gaucher-associated and idiopathic Parkinson’s models. The company is first pursuing Gaucher’s patients who develop Parkinson’s, with plans to expand into broader GBA-associated and idiopathic PD populations. Beyond CSM-101, Casma is building a pipeline that applies its lysosomal and autophagy platform to neurodegeneration, oncology, inflammation, and metabolic disorders. Casma recently secured $7.6 million in non-dilutive grant funding to complete biomarker development and IND-enabling studies for CSM-101, positioning the candidate for first-in-human trials. No additional financial metrics or revenue figures were disclosed.

Total raised

$162M

Funding rounds

4

Latest round

Equity

Latest activity

Mar 2026