Drug intelligence / Profile preview

Anc80AAV

Development stage
Preclinical
Lead developer
Eli Lilly
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Intracochlear
01

Overview

Anc80AAV is a **synthetic adeno-associated viral vector** developed as an inner-ear **gene therapy** delivery platform by Akouos and subsequently associated with Eli Lilly through acquisition. Rather than acting as a conventional small-molecule or biologic drug with intrinsic pharmacology, Anc80AAV functions as a rationally designed capsid for **delivery of therapeutic genetic cargo** to cochlear and vestibular target cells. Preclinical work has shown efficient transduction of multiple inner-ear cell types, including hair cells, after **intracochlear administration**, supporting its use as a precision genetic medicine platform for hereditary and other hearing or balance disorders. The design goal of the vector is to diverge from naturally occurring AAV serotypes and potentially mitigate limitations such as pre-existing anti-AAV immunity while enabling broad inner-ear gene transfer.

Other names
Anc80Anc-80Anc 80Anc80L65Anc-80L65Anc 80L65

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