Drug intelligence / Profile preview

AVB-401 (AviadoBio)

Development stage
Preclinical
Lead developer
Solid Biosciences
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathalamic
01

Overview

AVB-401 is an investigational gene therapy being developed by AviadoBio for the treatment of MAPT-related tauopathies. The therapy is designed to target the MAPT gene, which encodes the microtubule-associated protein tau, a protein that forms pathological aggregates in various neurodegenerative disorders. AVB-401 leverages AviadoBio's specialized delivery technologies, which may include their proprietary vMiX™ RNA silencing system and neuro-optimized delivery methods such as intrathalamic administration. These platforms are intended to achieve precise and widespread distribution of the therapeutic agent within the central nervous system to address the underlying genetic causes of tau-mediated neurodegeneration. As of early 2024, the program is in the preclinical stage of development.

02

Targets

MAPT (Microtubule-associated protein tau)

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