Drug intelligence / Profile preview

AAV.201

Development stage
Preclinical
Lead developer
Regeneron Pharmaceuticals
Modality
Gene Therapies
Administration
Intracochlear
01

Overview

AAV.201 is an investigational **AAV-based inner-ear gene therapy** program originally developed by Decibel Therapeutics, with rights later moving under Regeneron following its acquisition of Decibel. The program is being developed for **bilateral vestibulopathy** and is designed to **regenerate vestibular hair cells within the vestibule** to restore balance function. Public company descriptions characterize it as using a **supporting cell-selective or cell-specific promoter** to deliver one or more **regeneration-promoting transgenes** to target inner-ear cells; however, the exact therapeutic gene target and molecular payload have not been publicly disclosed in the sources reviewed. The program appears to have remained **preclinical / research-stage** with no clearly identified human clinical trial for AAV.201 found in the reviewed sources.

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