ALXN2220

ALXN2220 (AAV9-CTNT-COBAG3) is an investigational gene therapy being developed by Alexion, AstraZeneca's Rare Disease group, for the treatment of dilated cardiomyopathy (DCM) associated with mutations in the BCL2-associated athanogene 3 (BAG3) gene. The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector, which has a natural tropism for cardiac tissue, to deliver a codon-optimized version of the human BAG3 cDNA. Expression of the transgene is driven by a cardiac-specific troponin T (cTNT) promoter to ensure targeted activity within cardiomyocytes. BAG3 is a critical co-chaperone protein that facilitates protein quality control (proteostasis) and maintains the structural integrity of the sarcomere. Mutations in BAG3 lead to haploinsufficiency, resulting in protein aggregation, myofibrillar disintegration, and progressive heart failure. By restoring functional BAG3 levels, ALXN2220 aims to stabilize cardiac structure and improve heart function in patients with this genetic form of DCM.

Other names

AAV9-cTNT-BAG3AAV-9-cTNT-BAG3AAV 9-cTNT-BAG3BAG3 gene therapyBAG-3 gene therapyBAG 3 gene therapy