CRISPR-GNDM®

Payload packed into AAV to edit epigenome

CRISPR-GNDM® is a proprietary epigenome editing technology developed by Modalis that combines dCas9, which lacks cleavage enzyme activity, with an epigenome editor/modulator and a guide RNA (gRNA) that defines the target site in the genome. CRISPR-GNDM® is packaged in a single AAV and efficiently delivered to target cells to actuate therapeutic gene expression modulation.

CRISPR-GNDM® is packed into AAV and then delivered to target cells.

CRISPR-GNDM® is encoded as DNA and delivered by a AAV vector.
When CRISPR-GNDM® is delivered to a target cell, it is read out as a GNDM protein and the GNDM protein binds to the promoter region of the target gene and turns the target gene expression on/off. In the future, CRISPR-GNDM® may be delivered by other delivery technologies such as lipid nanoparticles.

However, AAV vectors are currently considered superior in terms of transfection efficiency and selectivity and are therefore used in combination with other delivery technologies such as nanocapsules.

CRISPR-GNDM® will bring about long-term medical care
The results of animal testing have shown that CRISPR-GNDM® is 1) administered in a single dose, 2) has a long-lasting effect, and 3) is capable of fundamentally improving the condition of the disease.

CRISPR-GNDM® can be easily optimized for use in a wide range of diseases.

The CRISPR-GNDM® technology consists of 3 main components: 1) a unit called guide RNA (gRNA), which is designed to uniquely bind to the switch part of the target gene, 2) dCas9, which is an enzyme-null version of Cas9, and 3) a modulator, which controls the expression level of the target gene. Since 2 and 3 are off-the-shelf, the only component we need to optimize for each target is 1, which is relatively easy to design and generate.