Intellia moves closer to clinic with CRISPR tech

Intellia Therapeutics has taken another step toward human trials of its gene editing technology after reporting new data in animal models.

The CRISPR specialist says it has been able to show for the first time that it is able to not only achieve long-term suppression of a gene using its gene-editing CRISPR/Cas9 drug, in this case the sequence coding for serum transthyretin (TTR) protein, but also demonstrate a dose-dependent reduction in the activity of the target gene in a second animal species.

New data from studies in mice show that a previously reported 97% reduction in TTR—driven by 70% gene editing efficiency working in mouse livers—lasts for up to six months from a single dose. Meanwhile, a study in rats showed a similar dose-dependent reduction in the TTR gene, and crucially evidence of comparable activity in a second species as Intellia builds the case to move to the clinic.

Read more here- Intellia moves closer to clinic with CRISPR tech