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DYNE-301
- Development stage
- Preclinical
- Lead developer
- Dyne Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody Conjugates → Antibody-Based Therapeutics
01
Overview
DYNE-301 is a therapeutic candidate developed by Dyne Therapeutics for the treatment of **Facioscapulohumeral Muscular Dystrophy (FSHD)**. It is an antisense oligonucleotide (ASO) conjugated to an antibody via a linker, utilizing Dyne's proprietary **FORCE platform**. The drug targets the **DUX4 gene**, aiming to reduce its expression in muscle cells, which is critical for addressing the genetic basis of FSHD. The FORCE platform is designed to enhance delivery of the ASO to muscle tissue.
- Other names
- FSHD ProgramDyne FSHD program
02
Targets
DUX4 (Double Homeobox 4)
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