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MDL-105

Development stage: Preclinical
Lead developer: Modalis Therapeutics
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies

Overview

MDL-105 is a preclinical-stage gene therapy developed by Modalis Therapeutics for the treatment of dilated cardiomyopathy (DCM). It utilizes the proprietary CRISPR-GNDM (guide nucleotide directed modulation) epigenome editing technology to upregulate the expression of the Titin (TTN) gene. The therapy is specifically designed for DCM caused by Titin gene truncating mutations (TTNtv), which can lead to decreased cardiac contractile force and heart failure. MDL-105 works by activating transcription of the TTN gene to restore normal levels of the Titin protein without introducing double-strand DNA breaks or altering the underlying DNA sequence.

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