AAV gene therapy with transferrin receptor-dependent capsid for ABCD1

AAV gene therapy with transferrin receptor-dependent capsid for ABCD1 is an experimental gene therapy designed for the treatment of X-linked Adrenoleukodystrophy (ALD). Developed by researchers at the Wyss Institute, Harvard Medical School, and Mass General Brigham, the therapy utilizes an engineered adeno-associated virus (AAV) capsid designed to bind the human Transferrin Receptor (hTfR). This binding facilitates receptor-mediated transcytosis across the blood-brain barrier (BBB), enabling widespread delivery of the functional *ABCD1* transgene to the central nervous system (CNS) following systemic intravenous administration. By enhancing BBB penetration, the vector aims to achieve therapeutic efficacy at significantly lower systemic doses than conventional vectors like AAV9, thereby potentially reducing risks of dose-dependent toxicities such as hepatotoxicity and dorsal root ganglia (DRG) toxicity. The program also explores the use of cell-type specific promoters, such as oligodendrocyte-restricted and microglial-restricted promoters, to optimize transgene expression and stability within disease-relevant CNS cell types.

Other names

AAV-hTfR-ABCD1AAV-hTfR-ABCD-1AAV-hTfR-ABCD 1