Drug intelligence / Profile preview

AAVrh10.alphaPA

Development stage
Preclinical
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous, Intracerebroventricular, Intracranial
01

Overview

AAVrh10.alphaPA is an experimental gene therapy using the adeno-associated virus serotype rh10 (AAVrh10) as a viral vector to deliver a therapeutic transgene. AAVrh10 is distinguished by its enhanced ability to transduce neurons and glial cells in the central and peripheral nervous system and efficiently cross the blood-brain barrier, making it of interest for treating neurological diseases and certain genetic disorders. The suffix “alphaPA” likely designates a proprietary or experimental therapeutic payload not explicitly described in the literature provided. AAVrh10-based therapies have been investigated preclinically for disorders such as lysosomal storage diseases and leukodystrophies, but "AAVrh10.alphaPA" itself does not correspond to any approved drug or major clinical-stage product as of September 2025[2][4].

02

Targets

Terminal galactose-containing cell-surface glycansIgG (Immunoglobulin G)Sulfo-LacNAc (Sulfated N-acetyllactosamine-containing glycan)

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