Drug intelligence / Profile preview

AAV-PN

Development stage
Preclinical
Lead developer
Latus Bio
Modality
Gene Therapies
Administration
Intrathecal
01

Overview

AAV-PN is a proprietary, engineered adeno-associated virus (AAV) pan-neuronal capsid variant developed by Latus Bio. It is designed to achieve broad, efficient, and highly specific gene delivery to neurons throughout the brain and spinal cord while limiting off-target expression in peripheral tissues, such as the liver and dorsal root ganglia. In preclinical studies in non-human primates, low-dose intracerebroventricular (ICV) administration of AAV-PN demonstrated widespread central nervous system (CNS) biodistribution and transgene expression, achieving approximately 40% to 60% neuronal transduction across the cerebral cortex and up to 40% transduction of spinal cord motor neurons. AAV-PN exhibits significantly higher CNS transgene expression compared to conventional AAV9, making it a promising platform for the development of gene therapies targeting various neurological and central nervous system disorders.

Other names
AAV pan-neuronalAAV-PN capsid variant

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