Genome Editing Tools for Therapeutic Discovery

Modalis Therapeutics utilizes its proprietary CRISPR-GNDM® technology to address genetic disorders by modulating gene expression rather than altering the DNA sequence itself. This innovative platform employs a nuclease-null Cas9 protein, which is fused with functional moieties such as transcription activators or suppressors, and directed to specific gene loci using guide RNA (gRNA). By manipulating the epigenetic regulation of genes, CRISPR-GNDM® can effectively turn genes on or off, thereby correcting issues related to excessive or insufficient gene expression. This approach is particularly advantageous for treating monogenic disorders, where the causative gene is known, allowing for a more predictable success rate in clinical trials.

The delivery of the CRISPR-GNDM® components is facilitated through adeno-associated virus (AAV) vectors, which are recognized for their safety and efficacy in gene therapy applications. This method allows for the targeted transduction of cells, ensuring that the therapeutic components reach the intended site of action. The technology's scalability is another significant benefit, as it can be adapted to target various diseases simply by modifying the gRNA sequences. Importantly, CRISPR-GNDM® avoids the risks associated with traditional genome editing techniques, such as double-strand breaks and off-target effects, making it a promising therapeutic modality for the treatment of genetic disorders.

United States

Modalis Therapeutics Corporation (Tokyo Mothers 4883) is developing precision genetic medicines through epigenetic gene modulation. Modalis is advancing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double- stranded DNA cleavage, gene editing or base editing. Modalis is headquartered in Tokyo with laboratories and R&D facilities in Cambridge,

Modalis Therapeutics

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