On-target
gene therapies

Our mission

We are developing next-generation gene editing medicines to cure rare genetic diseases at their roots by correcting inborn genetic errors directly in patients’ cells.

Our technology

Safety first. Our synergic core technologies increase the precision of genetic modifications and reduce the risks for patients. We bring gene editing to the next level of specificity, making it more ready for applications in the clinic.

We bring gene editing to
the next level of specificity.