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For genetic diseases, we are capable of using a guided RNA that enables Cas9 to cut DNA directly as a targeted site within a disease-causing gene. We can also cut DNA at a different site – a specific region responsible for regulating genes. This helps us ameliorate certain genetic defects through gene correction or disruption.

For cell therapies, specifically, we target genes that directly improve the efficacy and safety of the therapy when disrupted. Alternatively, we can insert new genes within cells to give them completely new abilities. Cells can be edited either in vivo or ex vivo – inside or outside the body, that is.

What does this mean, precisely?



The respective CRISPR-Cas9 components are packed within a ‘delivery vehicle’ – e.g. lipid nanoparticles. The therapeutic is then delivered either systemically or to a targeted organ like the liver.


Cells are first extracted from the patient. CRISPR-Cas9 is then transferred to the cells in culture, which produces the desired change or ‘edit’. Edited cells are finally injected back into the patient.

Through ‘precision medicine’, we can transform human healthcare and treat a range of unmet medical needs such as genetic diseases and disorders.

Near-term ex vivo applications, for example, can treat cancer and blood disorders. Today, there are many challenges and obstacles associated with cancer treatment such as immunosuppressive tumor microenvironment, manufacturing complexity, and poor infiltration of engrafted cells, etc. and these must be overcome for further treatment with different forms of cancer.

Recently, the antitumor activities of CAR‐T and TCR‐T cells have shown great improvement with the utilization of CRISPR-/Cas9 gene editing technology. Thus, the genome editing system could be a powerful genetic tool to use for manipulating T cells and enhancing the efficacy of cell immunotherapy.

Thanks to extensive research into the microbiome, we now understand the complex interplay between microbes and humans better than ever.

CRISPR holds a lot of promise for treating household pets via new cell therapies, through which certain genes and cells can be developed to cater to genetic diseases or conditions that your pets may be suffering from.

Life-saving products like vaccines, gene therapy vectors and recombinant therapeutic proteins are manufactured from major cell culture systems. With our scientific, we can improve these cell systems to yield not only higher qualities but also larger quantities of therapeutic biomaterials.