Biotech Entrepreneur Cathy Tie Pushes for Gene-Editing in Human Embryos to Prevent Disease

Cathy Tie, a biotech founder known as “Biotech Barbie,” is making a strong case for using gene-editing technology on human embryos to prevent serious inherited diseases. She argues that it is a moral duty to explore this option for families facing genetic conditions, even though the science remains risky and controversial.

Tie started her company, Origin Genomics, in the United States earlier this year. The company focuses on developing precise gene-editing methods to correct disease-causing mutations in embryos before pregnancy. So far, Origin is testing its tools in human embryonic stem cells donated for research, not in actual pregnancies.

The main approach involves advanced gene-editing techniques called base editing and prime editing, which allow scientists to make small, accurate changes to DNA. However, recent studies, including one from Columbia University, show that while these methods can be precise, they also carry risks like unintended genetic changes and mosaicism—where not all cells carry the same edit. These risks currently prevent safe use in clinics.

Experts warn that gene-editing in embryos is still not ready for real-world use because even thorough testing can’t guarantee that no harmful changes will be passed on to future generations. Embryo biopsies only sample a few cells, so some problems might go undetected until much later in life.

Regulatory hurdles also stand in the way. In the US, federal laws restrict funding and clinical trials involving heritable gene editing. The Food and Drug Administration oversees genetically modified cells but cannot approve clinical research on germline editing at this time.

Internationally, organisations like the World Health Organization and the US National Human Genome Research Institute say clinical use of gene-editing in embryos is irresponsible until safety and ethical concerns are fully addressed.

Tie believes that open, transparent research is the best way forward and that stopping work in this area could push it underground. She emphasises focusing on preventing serious diseases, not enhancing traits like intelligence.

While the promise of gene-editing to stop hereditary diseases is real, it comes with complex scientific, ethical, and legal challenges. For now, the technology is confined to research labs, with no clear timeline for clinical use.