Drug intelligence / Profile preview

9P801

Development stage
Unknown
Lead developer
Voyager Therapeutics
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Intravenous
01

Overview

9P801 is an engineered **adeno-associated virus capsid** developed by Voyager Therapeutics as a **gene therapy delivery vector** for central nervous system applications. Generated using Voyager's TRACER RNA-driven directed evolution platform, the capsid is designed to enable efficient **intravenous delivery across the blood-brain barrier** with broad CNS distribution and strong neuronal tropism. Preclinical non-human primate data described widespread brain transduction, including activity in regions such as the putamen, thalamus, globus pallidus, and brainstem, with substantially improved performance versus AAV9 and comparatively low liver transduction. 9P801 appears to be a platform delivery capsid rather than a fully specified therapeutic drug product, and no specific transgene, branded product name, or disclosed clinical indication has been identified from the provided context.

02

Targets

ALPL (Tissue-nonspecific alkaline phosphatase)

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