A summary of the guest presentation at the ISCT 3rd Annual Global Regulators Summit (6th May 2025, Ernest N. Morial Convention Center, New Orleans, USA) by Professor Henry (Hank) Greely, Director, Center for Law and the Biosciences, at Stanford University.
Rapporteur:
Patrick Foong, PhD
Western Sydney University
Sydney, NSW, Australia
In November 2018, at the second gene editing summit, Dr. He Jiankui announced the world’s first gene-edited twins (and a third embryo). He was regarded as both a hero and a villain. The Chinese government then changed the law he was found guilty and served three years in jail.
Dr. He is out of jail now. He continues gene editing research with no support from the Chinese government. Dr. He said the children are healthy (now about 6 years old), but this has not been verified. Interestingly, a Russian scientist, Denis Rebrikov, also announced plans to produce gene-edited babies, and Professor George Church (Harvard University) takes a more nuanced view than many scientists on Dr He‘s gene editing of human babies; even speaking in defence of Dr. He.
In the bioethics debate, there are three views. First, there are safety and efficacy issues. Several problems may arise, including off-target effects and mosaicism. Moreover, embryos cannot consent, and there are also concerns about commercialisation.
Next, even if germline editing technology is safe, this is considered by some as playing God. The risks are unknown. There is the precautionary principle, which encourages taking preventive action when there is potential for harm, even if scientific certainty about that harm is lacking. It is based on the principle of "first do no harm," as stated in the bioethics principle of non-maleficence, and applies to situations involving suspected risks to humans.
Finally, even if the technology is safe, germline editing should be conducted only for specific purposes, such as editing out diseases, but not for others, such as enhancement, producing super babies or designer babies.
It is also noted that there are equity issues related to access to technology, such as those between rich and poor countries.
The regulatory approaches are as follows:
- Do nothing, let it happen: this is not an attractive option. There are risks to the babies and could lead to medical tourism although probably not in the next 10-20 years.
- Regulate carefully: ensure it is regulated for safety and efficacy. But what is the drug? It does not clearly align with the definition of a drug in the US legislation (Food, Drug and Cosmetic Act).
- Ban it all: this approach is currently adopted in Europe but the majority of the American public opinion says “edit to prevent disease” although they may not understand heritability.
- Allow it: to allow it only for disease treatments, but not for enhancements. Even if this works well, is it a necessary alternative? Existing options, such as IVF and PGD, are available, but they have their own set of problems.
In Europe, there is a strict ban on germline editing. It is generally banned for clinical purposes. While research on germline editing is permitted in some countries, such as the UK, it requires licensing and strict regulations. The EU's regulatory and ethical framework for gene therapy is guided by the 2000 EU Charter of Fundamental Rights and the 1997 Convention on Human Rights and Biomedicine (Oviedo Convention). The Oviedo Convention prohibits interventions in the human genome that aim to introduce modifications in the genome of descendants.
In the UK, the Human Fertilisation and Embryology Authority (HFEA) needs to approve the research. HFEA regulates germline editing via the Human Fertilisation and Embryology Act 1990. While the Act includes provisions for germline cells, it does not explicitly mention germline gene editing. The HFEA has granted a license to research on human embryos using gene editing. Still, the study must be conducted within the first 14 days of embryo development, after which the embryos must be destroyed. The HFEA also prohibits transferring edited embryos into a woman for treatment under a research license.
In the USA, there is no active Investigational New Drug (IND) application. While there is no explicit federal law banning research in the area of germline editing, funding for such research is restricted. The FDA is not permitted to consider clinical trial applications that involve intentionally creating or modifying human embryos to include heritable genetic modifications.
In conclusion, this is not an urgent matter, and we are not likely to see it move to the clinic soon. There isn’t a good use case, there is political resistance, and a lack of monetary incentive. However, a “ban it all” response today provides opportunity for flexibility later – it is a matter of spending political capital. An IVF lab is required for germline gene editing, and some nonhuman primate work has been conducted in China. However, there has been no public work that has come close to human use, although it may be ongoing in secret.
#Regulatory