Synonyms: DNL-343
Compound class:
Synthetic organic
Comment: DNL343 is an investigational small molecule eIF2B activator [1]. It blocks activation of the integrated stress response (ISR), a biological process that plays a role in driving neurodegeneration in diseases such as ALS/MND and vanishing white matter disease (VWMD). DNL343 was designed to address ISR-mediated neurodegeneration.
The HGNC lists 4 human genes for eIF2B subunits (EIF2B1-EIF2B4). |
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Bioactivity Comments |
DNL343 demonstrates high CNS penetrance, and reduces ISR activation, premature mortality and biomarkers of neurodegeneration in in vivo models [1]. |