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BMN 293

Development stage: Unknown
Lead developer: BioMarin
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intravenous

Overview

BMN 293 (also known as DiNA-001) is an adeno-associated virus (AAV)-based gene therapy developed for the treatment of hypertrophic cardiomyopathy caused by mutations in the MYBPC3 gene. The therapy works through gene transference to replace or supplement myosin-binding protein C in cardiac muscle cells. It is being developed collaboratively by BioMarin Pharmaceutical and DiNAQOR AG. The drug targets MYBPC3-linked cardiomyopathy and aims to deliver therapeutic genes directly to heart tissue while minimizing exposure to other organs[1][2][3][4].

Other names: DiNA-001DiNA001DiNA 001DINA-001/ADINA-001/B

Targets

MYBPC3 (Myosin-binding protein C, cardiac-type)

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