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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No information available. |
Summary of Clinical Use |
DNL343 is in clinical development for ALS. |
Clinical Trials | |||||
Clinical Trial ID | Title | Type | Source | Comment | References |
NCT04297683 | HEALEY ALS Platform Trial - Master Protocol | Phase 2/Phase 3 Interventional | Massachusetts General Hospital | ||
NCT05842941 | HEALEY ALS Platform Trial - Regimen G DNL343 | Phase 2/Phase 3 Interventional | Massachusetts General Hospital | ||
NCT04268784 | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL343 in Healthy Volunteers | Phase 1 Interventional | Denali Therapeutics Inc. |