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MC-DX4
- Development stage
- Preclinical
- Lead developer
- Sanofi
- Modality
- miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous (expected; Preclinical Data Suggests Systemic Administration)
01
Overview
MC-DX4 is a preclinical-stage antibody-RNA conjugate (ARC) therapy developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a rare genetic muscle disorder. The drug consists of an antisense oligonucleotide or microRNA designed to specifically inhibit expression of the DUX4 gene, which is aberrantly expressed in FSHD and drives disease pathology. The RNA therapeutic component is conjugated to a muscle-targeted antibody—using proprietary chemistry—to enhance delivery and stability in muscle tissue. This targeted approach aims to selectively suppress DUX4 in affected muscles, potentially halting or reversing disease progression. MC-DX4 was discovered by miRecule using their genomics-based DREAmiR platform and is being co-developed with Sanofi[1][2][3][8].
02
Targets
DUX4 (Double Homeobox 4)
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