Drug intelligence / Profile preview

aquaporin-1 gene therapy

Development stage
Preclinical
Lead developer
Allegheny Health Network
Modality
Gene Therapies
Administration
Intralesional
01

Overview

**Ultrasound-assisted aquaporin-1 gene therapy** is an investigational, non-viral salivary-gland gene-transfer approach for **radiation-induced xerostomia** following head and neck cancer radiotherapy. The program combines a plasmid encoding human **aquaporin-1**, lipid microbubbles supplied by Lantheus, and low-frequency ultrasound delivered after direct parotid-gland injection. Ultrasound-driven microbubble oscillation produces sonoporation, transiently increasing salivary-gland cell-membrane permeability and enabling plasmid uptake. Expression of the aquaporin-1 water channel in surviving, radiation-resistant ductal cells is intended to restore transcellular water flux and saliva secretion. The approach is being developed by Allegheny Health Network with collaborators including the University of Michigan and Lantheus, and remains preclinical/IND-enabling rather than a registered human clinical-stage product.

Other names
AQP1 gene therapyAQP-1 gene therapyAQP 1 gene therapyultrasound-assisted aquaporin-1 gene therapyultrasound-assisted non-viral gene transfer of AQP1ultrasound-assisted non-viral gene transfer therapyUAGT
02

Targets

AQP1 (Aquaporin-1)

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