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A pioneer in Epigenome Editing

A pioneer in Epigenome Editing

The timing, location, and abundance of the approximately 20,000 proteins that compose our bodies are meticulously regulated, much like a symphony orchestra. This precise coordination is orchestrated by a gene control system known as the epigenome. When this system malfunctions, it can lead to disease. CRISPR-GNDM® offers the ability to selectively target and regulate the epigenomes of specific genes, enabling us to restore harmony and treat various ailments.

Genetic Disorders

Genetic disorders are often caused by errors or mutations in DNA sequence, making gene-level intervention essential to develop therapeutic cures. Gene therapies can rescue disease-associated mutations by delivering corrective DNA or RNA sequences through cell-targeting viral vectors and nanoparticles.

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CRISPR/Cas9 system

Genome editing is a new area of gene therapy, made possible by the development of CRISPR technology. CRISPR can precisely target faulty parts of our DNA, either cutting them out or inserting the correct genes, making genome editing a practical solution for treating genetic diseases.

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Epigenome Editing

Epigenome editing is a technology that regulates the expression of specific genes without altering the DNA sequence or causing double-strand breaks. It allows genes to be turned ON or OFF by Chemical modification of DNA and histones.

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CRISPR-GNDM Technology

CRISPR-GNDM® is an advanced epigenome editing technology developed by our company, designed to treat diseases by efficiently delivering a compact epigenome editor. This editor can be packed in AAV vectors for delivery to target cells, enabling the selective activation or suppression of specific genes with remarkable precision.

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BUSINESS

Multilayered deployment of hybrid in-house and collaborative development pipelines

The in-house pipelines are candidate therapeutics which the company develops using CRISPR-GNDM® technology with its own funding, and at a certain stage, the baton is passed to the partner for subsequent development and sales through a licensing agreement. Collaborative pipelines are ones in which the company develops therapeutic drugs using CRISPR-GNDM® technology with a partner company for a target that the partner selected or that both parties agree on. The partner then takes over the development later on.
PIPELINE

Muscular disorders

Cardiomyopathy

CNS disorders

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