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MDL-103
- Development stage
- Unknown
- Lead developer
- Modalis Therapeutics
- Modality
- Gene Therapies, Small Molecules
- Administration
- Intravenous
01
Overview
MDL-103 is an investigational AAV-based gene therapy developed by Modalis Therapeutics for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It utilizes Modalis's proprietary CRISPR-GNDM technology to epigenetically silence the DUX4 gene, which is aberrantly activated in FSHD and drives disease pathology. Unlike traditional genome-editing approaches that introduce double-strand DNA breaks, MDL-103 modulates gene expression without cutting DNA, aiming for durable suppression of DUX4 with muscle-specific delivery via an adeno-associated virus (AAV) vector. Preclinical studies have demonstrated promising efficacy in mouse models and safety in non-human primates[1][2][4][7][10].
- Other names
- MDL 103MDL103MDL-103CRISPR-GNDM gene editing therapy
02
Targets
DUX4 (Double Homeobox 4)
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