SRD-001

SRD-001 is an investigational gene therapy developed for the treatment of heart failure and cardiomyopathy, including in patients with Duchenne muscular dystrophy (DMD). It consists of an adeno-associated virus serotype 1 (AAV1) vector encoding the human SERCA2a gene. The therapy is designed to increase expression and functional activity of the sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), a protein critical for calcium handling in cardiac muscle cells. Downregulation of SERCA2a is implicated in impaired relaxation and contractility seen in various forms of heart failure. By delivering extra copies of the SERCA2a gene directly to cardiac ventricular muscle cells via intracoronary infusion, SRD-001 aims to restore normal calcium cycling, improve myocardial relaxation and contraction, and ultimately enhance cardiac function. The drug has received FDA fast track designation and orphan drug status for its lead indications[1][3][4][8].

Other names

Sardocor SERCA2a gene therapySRD-001SRD001SRD 001