ALXN2310

ALXN2310 is an investigational gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver a functional copy of the *BAG3* (BCL2-associated athanogene 3) gene to cardiomyocytes. It is being developed by Alexion, the rare disease unit of AstraZeneca, for the treatment of dilated cardiomyopathy (DCM) associated with mutations in the *BAG3* gene. BAG3 is a critical co-chaperone protein that plays a vital role in maintaining protein homeostasis (proteostasis) and structural integrity within heart muscle cells by facilitating the degradation of misfolded proteins and stabilizing the sarcomere at the Z-disc. Mutations leading to BAG3 deficiency result in protein aggregation, myofibrillar disruption, and progressive heart failure. By restoring BAG3 expression, ALXN2310 aims to improve cardiac function and prevent disease progression. This program was acquired by AstraZeneca from Pfizer's rare disease gene therapy portfolio in 2023.

Other names

AAV9 BAG3 gene therapyAAV-9 BAG3 gene therapyAAV 9 BAG3 gene therapyBAG3 gene therapyBAG-3 gene therapyBAG 3 gene therapy