Human Receptor 1

Human Receptor 1 is a conserved transmembrane protein identified as the primary mediator for the blood-brain barrier (BBB) crossing of Sangamo Therapeutics' STAC-BBB (CNSRCV300) AAV capsid. It is highly expressed on brain endothelial cells and facilitates the entry of therapeutic payloads into the central nervous system through receptor-mediated transcytosis. The receptor exhibits high-affinity binding to the STAC-BBB capsid in the low picomolar range, a property that is conserved across humans, non-human primates, and mice. This interaction enables widespread and robust transgene expression throughout the brain, which is critical for the delivery of genomic medicines such as Zinc Finger Repressors. In clinical development, this target is leveraged to treat fatal neurodegenerative conditions like prion disease by allowing for systemic administration of brain-penetrant therapies. While its specific molecular identity is often kept proprietary in early disclosures, it is distinguished from other common BBB targets like the transferrin receptor (TfR1) and alkaline phosphatase (ALPL).

Other names

Receptor 1STAC-BBB receptorCNSRCV300 receptorPutative BBB receptor 1

Facilitates the transport of engineered AAV capsids across the blood-brain barrier via receptor-mediated transcytosis (RMT) upon high-affinity binding to the capsid surface.