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DINA-003
- Development stage
- Preclinical
- Lead developer
- DiNAQOR
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intracoronary
01
Overview
DINA-003 is an investigational gene therapy candidate being developed by DiNAQOR for the treatment of hypertrophic cardiomyopathy (HCM). HCM is a genetic heart condition characterized by abnormal thickening of the heart muscle, which can lead to heart failure and sudden cardiac death. DINA-003 is currently in the discovery phase and is designed to utilize DiNAQOR's proprietary cardiac-targeted gene therapy platform, which typically employs engineered adeno-associated virus (AAV) vectors to deliver therapeutic transgenes directly to the myocardium. While DiNAQOR's lead program, DINA-001, targets mutations in the *MYBPC3* gene, DINA-003 represents a separate program aimed at addressing HCM through an undisclosed genetic target or therapeutic mechanism.
- Other names
- DINA 003DINA003DINA-003
02
Targets
MYBPC3 (Myosin-binding protein C, cardiac-type)
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