STAC-BBB

STAC-BBB is a novel, engineered adeno-associated virus (AAV) capsid developed to enable robust penetration of the blood-brain barrier (BBB) and widespread transduction of neurons throughout the central nervous system (CNS). It was created using Sangamo Therapeutics' SIFTER capsid platform and demonstrates 700-fold higher neuronal transgene expression compared to benchmark AAV9 in non-human primate studies. The capsid is designed for intravenous administration, showing strong CNS enrichment and de-targeting from peripheral organs such as the liver and dorsal root ganglia. Its primary application is as a delivery vehicle for genomic medicines—especially zinc finger-based epigenetic regulators—to treat neurological diseases including prion disease, tauopathies, Alzheimer’s disease, Parkinson’s disease, ALS, Huntington’s disease, Friedreich’s ataxia, and spinocerebellar ataxias. Preclinical data indicate favorable safety with no significant toxicity in brain or peripheral tissues at clinically relevant doses[1][3][6][8].