Drug intelligence / Profile preview

AAV6-NFIB

Development stage
Preclinical
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous
01

Overview

AAV6-NFIB is an adeno-associated virus serotype 6 (AAV6) mediated gene therapy designed to overexpress the transcription factor Nuclear Factor I-B (NFIB). In the context of liver fibrosis and metabolic dysfunction-associated steatohepatitis (MASH), NFIB is typically downregulated in hepatic stellate cells (HSCs), which are the primary drivers of fibrogenesis. By restoring NFIB expression, this therapy targets the promoter region of Chemokine C-C motif ligand 5 (CCL5), directly suppressing its transcription. This inhibition of the NFIB/CCL5 axis reduces oxidative stress and prevents the activation and differentiation of HSCs, thereby mitigating the progression of liver fibrosis and offering a potential therapeutic approach for chronic liver diseases.

Other names
AAV6-Nuclear Factor I-BAAV-6-Nuclear Factor I-BAAV 6-Nuclear Factor I-B
02

Targets

NFIB (Nuclear factor I-B)HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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