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TRPML1 Agonist
- Development stage
- Preclinical
- Lead developer
- Casma Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Casma Therapeutics is developing a small molecule agonist of the TRPML1 (Mucolipin-1) channel as a therapeutic approach for central nervous system (CNS) diseases. TRPML1 is a lysosomal cation channel that plays a critical role in the autophagy-lysosome pathway by regulating lysosomal calcium release, which is essential for vesicle trafficking, lysosomal acidification, and fusion with autophagosomes. By pharmacologically activating TRPML1, Casma aims to enhance the cellular clearance of toxic protein aggregates and damaged organelles, thereby addressing the underlying pathology of neurodegenerative conditions such as Alzheimer's disease, Parkinson's disease, and various lysosomal storage disorders. The program is currently in the discovery and preclinical development stages.
- Other names
- TRPML1 Agonist CNS ProgramTRPML-1 Agonist CNS ProgramTRPML 1 Agonist CNS ProgramTRPML1 AgonistTRPML-1 AgonistTRPML 1 Agonist
02
Targets
MCOLN1 (Transient receptor potential cation channel mucolipin subfamily member 1)
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