Drug intelligence / Profile preview

DYN-T0

Development stage
Preclinical
Lead developer
Dynomics
Modality
Gene Therapies
Administration
Intravenous
01

Overview

DYN-T0 is an investigational gene therapy being developed by Dynomics for the treatment of cardiomyopathies associated with the phospholamban (PLN) R14del mutation. The therapy utilizes Dynomics' proprietary AJuV (Adeno-associated Virus for Unmet Cardiovascular needs) platform, which is engineered to provide enhanced cardiac tropism and efficient delivery of therapeutic payloads to the heart. The PLN-R14del mutation is a genetic variant that leads to impaired calcium handling in cardiomyocytes, resulting in dilated cardiomyopathy, arrhythmogenic cardiomyopathy, and progressive heart failure. DYN-T0 aims to address the underlying genetic cause of the disease by delivering a therapeutic genetic payload to cardiac tissue to restore proper calcium cycling. It is currently in the discovery and lead optimization stage.

02

Targets

PLN (Phospholamban)

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