RP-A601 (Rocket Pharmaceuticals, Inc.)

RP-A601 is an adeno-associated virus (AAV) serotype 9-based gene therapy designed to deliver a functional copy of the BCL2-associated athanogene 3 (BAG3) gene to the heart. BAG3 is a co-chaperone protein essential for maintaining cardiomyocyte proteostasis, structural integrity of the sarcomere, and calcium signaling. Mutations in the BAG3 gene lead to haploinsufficiency, resulting in dilated cardiomyopathy (DCM). By restoring BAG3 protein levels, the therapy aims to improve cardiac function and prevent disease progression in patients with BAG3-associated DCM. It was originally developed by Renovacor, which was subsequently acquired by Rocket Pharmaceuticals. The therapy is currently being evaluated in clinical trials using a one-time intracoronary infusion to target the myocardium.

Other names

BAG3-DCM gene therapyBAG-3-DCM gene therapyBAG 3-DCM gene therapyAAV9-BAG3 gene therapyAAV-9-BAG3 gene therapyAAV 9-BAG3 gene therapy