Drug intelligence / Profile preview

SRD-064

Development stage
Unknown
Lead developer
Sardocor
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Inhalation, Intravenous
01

Overview

SRD-064 is an adeno-associated virus (AAV) vector-based gene therapy being developed by Sardocor for the treatment of pulmonary hypertension. The therapy is designed to deliver a functional copy of the ATP2A2 gene, which encodes the Sarcoplasmic/Endoplasmic Reticulum Calcium ATPase 2a (SERCA2a) enzyme. SERCA2a is a critical regulator of calcium homeostasis in cardiomyocytes and vascular smooth muscle cells; its downregulation is a hallmark of pulmonary hypertension and associated right heart failure. By restoring SERCA2a expression, SRD-064 aims to improve pulmonary vascular remodeling, decrease pulmonary artery pressure, and enhance right ventricular contractility. The program is currently in Phase 1 clinical development.

02

Targets

SERCA2

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