The USA-based company is applying novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation.

In June 2023, the company emerged from stealth mode with a $51 million Series A financing. In addition, the two-year old start-up granted Japanese drug major Astellas Pharma (TYO: 4503) an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM).