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del-brax
- Development stage
- Unknown
- Lead developer
- Novartis
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
DEL-BRAX (DWH213) is an investigational small interfering RNA (siRNA) therapeutic candidate developed by Hefei China Resources Novartis Pharmaceutical for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The drug functions by targeting and silencing the DUX4 (Double Homeobox 4) gene, whose aberrant expression in skeletal muscle is the primary driver of FSHD pathogenesis. By utilizing the RNA interference (RNAi) pathway to degrade DUX4 mRNA, DEL-BRAX aims to suppress the production of the toxic DUX4 protein, thereby preventing muscle cell death and halting disease progression. The asset is currently reported to be in Phase 3 clinical development.
- Other names
- DEL-BRAX
02
Targets
TFRC (Transferrin Receptor)DUX4 (Double Homeobox 4)
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