Kano Therapeutics' Gene-Editing Breakthrough: A New Approach to Genetic Medicine

To our immune system, a potentially lifesaving gene therapy can look a lot like a dangerous infection. That's because most genetic medicine uses viruses or double-stranded DNA to deliver genetic information to target cells. DNA in its traditional double helix form can lead to toxic immune stimulation and be difficult to package into cellular delivery vehicles. As a result, the reach of genetic medicine is limited today. Kano Therapeutics is taking a different approach to genetic therapies. The company is developing gene-editing technologies using circular single-stranded DNA (cssDNA), a biomolecule that is less toxic than double stranded DNA and more stable than RNA, and could be delivered more efficiently to many parts of the body to treat genetic diseases, cancers, and more. The company, which was founded by former MIT postdoc Floris Engelhardt, professor of biological engineering Mark Bathe, and John Vroom MBA '22, is developing a platform for manufacturing cssDNA of customized lengths and sequences, which could deliver genetic material to fix or replace faulty genes. "We can work with CRISPR and other gene-editing technologies," Engelhardt says. "CRISPR finds a location in a genome, binds to it, and cuts at that location. That allows you to edit a gene or stop a gene from functioning. But what if you have a loss-of-function disease where you need to insert a new piece of genetic code? Our approach allows you to replace whole genes or add genetic information."