Drug intelligence / Profile preview

AAV6.2

Development stage
Preclinical
Lead developer
Tulane University
Modality
Gene Therapies
Administration
Inhalation, Intranasal
01

Overview

AAV6.2 is a modified adeno-associated virus (AAV) serotype 6 vector optimized for enhanced transduction of airway epithelial cells. It is primarily utilized as a delivery vehicle in lung-directed gene therapies for genetic respiratory disorders, including cystic fibrosis, primary ciliary dyskinesia, and alpha-1-antitrypsin deficiency. Research indicates that while pre-existing systemic antibodies have limited impact on initial mucosal delivery, the combination of systemic humoral and cellular immunity can trigger T-cell mediated rejection of the transgene, posing a significant barrier to long-term expression in the lungs.

Other names
adeno-associated virus 6.2
02

Targets

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

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