Drug intelligence / Profile preview

CereAAV.YN

Development stage
Preclinical
Lead developer
Takara Bio
Modality
Gene Therapies
Administration
Intravenous
01

Overview

CereAAV.YN is an engineered adeno-associated virus (AAV) vector derived from the AAV2 serotype, developed by Takara Bio for the treatment of central nervous system (CNS) disorders. It was created by introducing specific amino acid substitutions into the CereAAV capsid, a variant identified through directed evolution of a random peptide-inserted AAV2 library. CereAAV.YN is designed to cross the blood-brain barrier (BBB) efficiently following systemic administration, addressing the limitations of traditional AAV2 vectors that require invasive direct intracerebral injection. Preclinical studies in mouse models demonstrated a 240-fold increase in gene transduction compared to AAV9, and studies in non-human primates (Cynomolgus macaques) showed successful gene delivery to over 70% of neuronal cells in the brain and spinal cord.

02

Targets

VPS35 (VPS35 (retromer complex))VPS29 (Vacuolar protein sorting-associated protein 29)

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