Key Takeaway:
- Cathy Tie advocates advancing embryo gene editing to prevent inherited diseases.
- Researchers warn that unintended genetic changes and long-term risks remain unresolved.
- U.S. funding and regulatory restrictions currently limit clinical development of heritable embryo editing.
Biotech founder Cathy Tie is urging regulators and researchers to advance gene editing of human embryos, arguing it could prevent inherited diseases despite unresolved safety risks.
Founder Calls Embryo Editing A Moral Imperative
Tie, founder of New York City-based Origin Genomics, argues in a new Trends in Genetics commentary that governments should provide funding and establish regulatory pathways for developing heritable embryo editing.
Gene Editing Of Human Embryos could allow doctors to correct disease-causing mutations before an embryo is transferred for pregnancy. Because genetic changes could pass to future generations, however, researchers must establish that editing does not create unintended changes or other harms.
“There are cautionary tales of first human trials where there was really no flashing red light, and people died,” said Hank Greely, a Stanford University law professor and director of its Center for Law and the Biosciences.
The debate follows Chinese scientist He Jiankui’s 2018 announcement that he had edited embryos that resulted in the birth of twins. His work drew international condemnation and led to criminal punishment in China.
Supporters say embryo editing could address cases in which existing reproductive options provide too few disease-free embryos. Ian Watts, a California engineer with Charcot-Marie-Tooth disease, and his partner, Cheyenne Ziegler, produced eight chromosomally normal embryos through three IVF rounds, but only three lacked the genetic variant associated with his disease.
“The choices currently available are either not having children or doing lots of IVF,” Watts said.
New Research Shows Promise But Leaves Safety Questions
Recent research into Gene Editing Of Human Embryos has demonstrated that base editing can make precise genetic changes, but the findings have not established that the technology is safe for pregnancy.
A Columbia University-led study published in Nature in September examined base editing at the PCSK9 and HBG genes. Researchers found high editing efficiency but also observed mosaic off-target activity, chromosomal abnormalities, and other unwanted effects. The researchers concluded that such consequences currently prevent clinical reproductive use.
Another Nature study published in June used base editing to investigate the role of the NANOG gene in human embryonic development. The work demonstrates the technique’s research potential but does not establish embryo editing as a reproductive treatment.
“Every mutation will therefore be a new medicine,” said Dieter Egli, who led the Columbia research, according to the report.
Tie says Origin is working to optimize base- and prime-editing methods using embryos donated for research, while acknowledging that the technology is not ready for human patients.
Researchers also caution that embryo testing cannot guarantee that every cell is free of unintended genetic changes. Some effects could remain undetected until later in life or potentially appear in future generations.
Regulators Face Barriers Before Human Trials
In the United States, federal restrictions remain a major obstacle. NIH policy prohibits federal funding for creating human embryos for research and says the agency will not fund gene-editing technologies in human embryos.
Federal law also restricts FDA action on applications involving embryos intentionally created or modified to contain heritable genetic changes. The fiscal 2026 Agriculture-FDA appropriations legislation includes a provision barring the use of funds to acknowledge certain submissions involving such modifications.
Some researchers say extensive testing must precede any clinical trial. Paula Amato of Oregon Health & Science University said studies should first establish safety in embryos and then progress through animal studies, including examination across generations.
Public attitudes are also part of the debate. A 2026 Ipsos survey of 8,688 respondents across the United Kingdom, Netherlands, Spain and Italy found that more respondents supported than opposed embryo genome editing in scenarios involving research and prevention of severe or life-threatening conditions.
Tie argues that preventing inherited disease should be considered part of reproductive choice, while opposing concerns center on safety, regulation, and the possibility of broader applications.
The central question remains whether researchers can demonstrate enough safety for changes that could affect not only one child but that person’s descendants.
