Drug intelligence / Profile preview

GNR-097

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

Overview

GNR-097 is an investigational gene therapy developed by AO GENERIUM for the treatment of Duchenne muscular dystrophy (DMD). The drug utilizes a recombinant adeno-associated viral vector of serotype 9 (AAV9) to deliver a truncated version of the dystrophin gene, known as micro-dystrophin, into muscle cells. This approach is designed to restore functional dystrophin expression and address the underlying genetic cause of the disease, which is characterized by progressive muscle wasting due to mutations in the DMD gene. GNR-097 is currently in Phase 1/2 clinical development.

Targets

DMD (Dystrophin)

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