Drug intelligence / Profile preview

rAAV2.5T

Development stage
Preclinical
Lead developer
University of Alabama at Birmingham
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intratracheal, Inhalation
01

Overview

rAAV2.5T is a recombinant adeno-associated virus (AAV) vector engineered for enhanced gene delivery to the respiratory tract. It utilizes a chimeric capsid derived from AAV2 and AAV5, specifically modified to improve apical tropism in human airway epithelium. In preclinical studies using ferret models, rAAV2.5T has demonstrated broad transduction across various airway epithelial cell types, including ciliated cells, goblet cells, basal cells, and ionocytes in the proximal airways, as well as club cells and alveolar type I (AT1) cells in the distal lung. The vector is being investigated as a delivery vehicle for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene to treat cystic fibrosis and other diffuse pulmonary diseases. It is often formulated with proteasome inhibitors like doxorubicin to further enhance transduction efficiency.

Other names
recombinant adeno-associated virus 2.5T
02

Targets

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

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