Proteostasis Meet Preclinical Milestone for Neurodegenerative Disease

Proteostasis Therapeutics, a biotechnology company focused on therapeutics targeting protein chaperone and proteasomal degradation pathways, announced achievement of a major preclinical milestone in drug development for neurodegenerative diseases. In collaboration with Biogen Idec, the company is developing compounds that enhance proteasomal activity by inhibiting Usp14, a deubiquitinating enzyme that targets several key proteins implicated in neurodegenerative diseases. The company successfully completed its preclinical milestone by demonstrating that these Usp14 inhibitors successfully clear aggregation-prone proteins in a neurodegenerative disease animal model. Is ALS on the table? Since Usp14 stabilizes TDP-43 protein (Lee et. al., 2010), there is evidence that these could be effective at clearing pathological aggregates characteristic of the disease.

Proteostasis Therapeutics, a biotechnology company focused on therapeutics targeting protein chaperone and proteasomal degradation pathways, announced achievement of a major preclinical milestone in drug development for neurodegenerative diseases. In collaboration with Biogen Idec, the company is developing compounds that enhance proteasomal activity by inhibiting Usp14, a deubiquitinating enzyme that targets several key proteins implicated in neurodegenerative diseases. The company successfully completed its preclinical milestone by demonstrating that these Usp14 inhibitors successfully clear aggregation-prone proteins in a neurodegenerative disease animal model. Is ALS on the table? Since Usp14 stabilizes TDP-43 protein (Lee et. al., 2010), there is evidence that these could be effective at clearing pathological aggregates characteristic of the disease.

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