Drug intelligence / Profile preview

BL001

Development stage
Preclinical
Lead developer
Bloom Science
Modality
Small Molecules
Administration
Oral
01

Overview

BL001 is a pharmacological agonist of LRH-1/NR5A2 (Liver Receptor Homolog-1 / Nuclear Receptor Subfamily 5 Group A Member 2) being investigated for its potential in treating type 1 diabetes (T1D). It works by activating LRH-1/NR5A2, which leads to a genetic and immunometabolic reprogramming of T1D immune cells. This reprogramming reduces pro-inflammatory markers and cytokine secretion, enhances mitohormesis in M1 macrophages, and promotes mitochondrial turnover in mature dendritic cells, shifting the immune response from pro-inflammatory to anti-inflammatory/tolerogenic. BL001 also inhibits CD4+ and CD8+ T-cell proliferation, increases regulatory T-cells (CD4+/CD25+/FoxP3+) and Th2 cells, and protects beta cells from apoptosis, thereby supporting their viability and maintaining insulin expression. This mechanism aims to attenuate autoimmune attack and promote beta cell survival/regeneration in T1D.

02

Targets

NR5A2 (Purinergic receptor P2X5)

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