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Find Clinical Drug Pipeline Developments & Deals by Caraway Therapeutics
Through the acquisition, Merck gains Caraway's pipeline of novel, small-molecule therapeutics for the treatment of genetically defined neurodegenerative and rare diseases.
TRPML1 is a non-selective cation channel that contributes to the regulation of lysosomal ionic contents and mediates communication with the rest of the cell.
TRPML1 normally functions to regulate and maintain the ionic composition in the lysosome, and mutations in TRPML1 lead to Mucolipidosis Type IV, which is typified by lysosomal dysfunction and toxic accumulation of proteins and lipids that have not been efficiently recycled.
TRPML1, a member of the transient receptor potential ion channel superfamily, targeting the lysosomal cation channel for the treatment of GBA-PD and other neurodegenerative diseases.
The Company will use the grant funding to further evaluate these effects and characterize potential biomarkers which may be used in future clinical trials of TRPML1 activators.
Collaboration to focus on development of TMEM175 modulators in Parkinson’s disease and other neurodegenerative disorders. Leverages Caraway’s unique drug discovery platform and expertise in cellular clearance.