Drug intelligence / Profile preview

bafilomycin A1

Development stage
Preclinical
Lead developer
Streptomyces griseus (natural product origin)
Modality
RNA-Targeting Small Molecules → Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
In Vitro (laboratory Use)
01

Overview

Bafilomycin A1 is a toxic macrolide antibiotic derived from Streptomyces griseus. It is the most widely used member of the bafilomycins and functions primarily as a potent and selective inhibitor of vacuolar-type H+-ATPase (V-ATPase). By inhibiting V-ATPase, Bafilomycin A1 prevents acidification within lysosomes and endosomes, thereby blocking autophagic flux at the late stage by preventing fusion between autophagosomes and lysosomes. This inhibition disrupts protein degradation, impairs cholesterol trafficking in lysosomes, and can induce apoptotic cell death in various cancer cells. Bafilomycin A1 has been used extensively as a research tool to study autophagy, endosomal acidification, viral infection mechanisms (including HIV-1 and SARS-CoV-2), multidrug resistance reversal in cancer cells, and other cellular processes dependent on vesicular acidification[3][4][5][6][7][8].

Other names
bafilomycin A1
02

Targets

V-ATPase (Vacuolar-type H+-ATPase)

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