Drug intelligence / Profile preview

rAAV4

Development stage
Preclinical
Lead developer
University of Kansas Medical Center
Modality
Gene Therapies
Administration
Intravenous
01

Overview

rAAV4 (recombinant adeno-associated virus 4) is a gene therapy vector belonging to Clade G. It is distinguished by its use of CD164, a type I transmembrane sialomucin, as an essential entry receptor, allowing it to transduce cells via a mechanism independent of the canonical AAV receptor (AAVR/KIAA0319L). In preclinical murine models, systemic administration of rAAV4 demonstrates a strong tropism for the lungs. The identification of CD164 as its primary receptor provides a molecular basis for engineering and optimizing Clade G AAV vectors for targeted in vivo gene delivery applications.

Other names
recombinant adeno-associated virus 4AAV4AAV-4AAV 4
02

Targets

CD164O-sialoglycans (Cell-surface O-linked sialoglycans)

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