JBI is a pre-clinical stage pharmaceutical company developing small molecules to modulate acetylation in the endoplasmic reticulum (ER) to restore proteostasis for treatment of Alzheimer’s disease (AD).
Target Cellular Machinery Within the Endoplasmic Reticulum (ER) to Restore Proteostasis
The ER is the organelle where most membrane and secreted proteins of the cell are synthesized, folded, and processed. It is also an important part of the cell’s proteostatic machinery. Proteostasis refers to the process by which the cell controls the synthesis, maturation, folding, trafficking and degradation of intra- and extracellular proteins. Age-related dysregulation of the proteostatic system is a root cause of many neurodegenerative diseases, including AD. JBI is the first company to demonstrate that inhibiting specific ER acetylation and restoring proteostasis has a neuroprotective effect in models of AD.
Our research program will produce novel compounds that modulate ER acetylation of a group of proteins that have been shown to be at the heart of AD pathogenesis. Our lead program stimulates the degradation of intracellular amyloid aggregates via autophagy while lowering cellular production of pTau, amyloid-β, and BACE1.