Drug intelligence / Profile preview

AAVDJ-CAG-hFIX

Development stage: Preclinical
Lead developer: Yecuris
Modality: Gene Therapies
Administration: Intravenous

Overview

AAVDJ-CAG-hFIX is a recombinant adeno-associated virus (rAAV) gene therapy vector utilizing the synthetic hybrid AAV-DJ capsid. It contains a CAG promoter driving the expression of human coagulation Factor IX (hFIX). AAV-DJ is a chimeric capsid engineered via DNA family shuffling of multiple wild-type AAV serotypes, offering high transduction efficiency in hepatocytes and resistance to immune neutralization. AAVDJ-CAG-hFIX is primarily used in preclinical research, such as in liver-humanized mouse models, to evaluate the efficacy of gene therapies for Hemophilia B by restoring circulating human Factor IX levels and correcting blood clotting defects.

Other names: AAV-DJ-CAG-hFIXAAV-DJ-CAG-human Factor IXAAVDJ-CAG-human Factor IX

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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