How should human genome editing be regulated?

How should human genome editing be regulated?

Two reports published last week by the World Health Organisation (WHO) provide the first global recommendations on appropriate institutional, national, regional and global governance mechanisms for human genome editing. Having had the privilege of participating in the consultation process, we now take a look at the recommendations.


A need for robust oversight

Human genome editing has enormous potential for treating and preventing disease. Many clinical trials based on the CRISPR-Cas9 system, for which the 2020 Nobel prize in chemistry was awarded, are already underway. For example, in June 2021, a landmark study showed a single IV transfusion of CRISPR components to patients suffering from the life threatening disease transthyretin amyloidosis, resulted in up to 96% reduction of the disease causing protein.

There is however, as the reports put it, a “gap in scientific understanding” of the risks associated with human gene editing, including for example the impact of off-target editing and long term effects. There are also significant ethical and social issues to consider, for example in the area of heritable genome editing (edits that could be passed on to subsequent generations) and editing to enhance or change human traits (e.g. for aesthetic purposes) or even add non-human traits.

Recognising a need for “robust oversight” in this area, the World Health Organization (WHO) tasked an expert advisory committee (the Committee) to advise and make recommendations on appropriate institutional, national, regional and global governance mechanisms for human genome editing.

Kilburn & Strode’s involvement with WHO’s expert advisory committee

How did the Committee go about its work? In short - very thoroughly! They spent two years reviewing the literature and consulting with a wide range of individuals and organisations, including the UN, academies of science and medicine, various professional bodies, and patient groups. We are delighted to have had the opportunity to take part in this process by presenting to the Committee on patents and genome editing in November 2020.


What does the first WHO committee report recommend?

The first report provides recommendations on governance and oversight in nine areas, focussing on improving systems to help ensure that human genome editing is used safely, effectively, and ethically.

The first of these recommendations calls upon the WHO itself to demonstrate moral and scientific leadership by making clear statements on the opportunities and challenges inherent to human genome editing, especially on ethical aspects. A particular recommendation is to reiterate the Director-General’s statement of July 2019, which strongly discouraged regulatory authorities from allowing work on clinical applications of human germline genome editing until its implications have been properly considered. While the WHO cannot stipulate global policy, the report argues that policy statements by the WHO, calling out what should or should not happen, can be effective, citing the recent example of statements on access to COVID-19 vaccines.

Another recommendation relates to clinical trial registries for human genome editing. In August 2019, following an interim recommendation of the Committee, the WHO launched a global human genome editing registry (Registry). This is about providing public access to information on clinical trials. But the Committee caution that careful monitoring is required, pointing to historical abuse of clinical trial registries in the field of stem cell research where unsanctioned procedures were given an air of legitimacy by their inclusion in clinical trials registries. Recommendations therefore include thorough review and approval by the appropriate research ethics committee before inclusion in the Registry, regular monitoring of the Registry by an expert committee and developing and reviewing a set of international standards for clinical trials involving human genome editing.

A third recommendation of interest is to establish a mechanism for confidential reporting of concerns about possibly illegal, unregistered, unethical and unsafe human genome editing research and other activities. Here, the Committee explicitly refers to the infamous “CRISPR-baby scandal”, where He Jiankui edited human embryos resulting in live births, following which he was sentenced to three years in prison in China. The Committee noted a need for support and protection for those reporting wrongdoing, suggesting a trustworthy international network of existing organisations is the way forward. In the meantime, the Committee suggests that concerns should be reported to the WHO, who would then share the information with the appropriate authorities.


What does the second WHO committee report recommend?

The second report sets out a framework for governance. The aim is to provide those tasked with strengthening oversight methods with the tools to help and the questions to consider. This is an extensive document divided into six parts. The Committee starts with a definition of good governance, then identifies particular challenges in different areas of human genome editing (including heritable genome editing and genetic enhancement), before moving on to a review of the tools, institutions and process of for governance (e.g. legislation, judicial rulings, conditions on research funding).

A particularly illuminating part of the report is a set of seven hypothetical scenarios, which help put everything in context. One such scenario envisages an international research team carrying out a clinical trial in West Africa to treat sickle cell disease, another looks at editing to enhance athletic ability, and perhaps one of the most ethically controversial scenarios looks a maverick researcher planning to alter the physical appearances of offspring using genome editing. In each case, the Committee highlights important questions to consider when developing oversight measures and sets out the tools, institutions and processes that could be involved. While the Committee noted that the scenarios are not all equal in terms of timescale or likelihood, they do provide a fascinating glimpse at the questions and challenges society may face in the coming years.


Do the WHO reports mention patents?

Yes! Steering clear of the well-documented CRISPR patent wars, the reports recognise the importance of patents in incentivising research and development, and as a possible form of governance. The Committee recommends that the WHO should encourage relevant patent holders to help ensure equitable access to human genome editing interventions. The WHO is also urged to encourage industry to build capacity for human genome editing in resource constrained countries, and to encourage dialogue between organisations such as WIPO and the WTO and patent holders to explore the potential for the adoption of appropriate ethical licensing requirements. Ethical licencing refers to the ability of a patent holder to set licencing conditions that prevent the protected invention being used for unethical purposes. As noted by the Committee, in comparison to legislative efforts, this could provide a more efficient governance mechanism.


What next?

The immediate next steps for the WHO, according to its press release, are to: 

  • Consider the next steps for the Registry (e.g. how to improve monitoring);

  • Facilitate development of a mechanism for confidential reporting of illegal, unregistered, unethical and unsafe human genome editing research; and

  • Lead webinars on regional/local needs, work towards building an inclusive global dialogue on frontier technologies, and create web-based resources for reliable information on human genome editing. 

We welcome these well thought-out and timely reports, and particularly the recognition of a role that patents and patent holders can play in the development of governance mechanisms. We look forward to seeing the recommendations being put into practice so that society can maximise the benefits of human genome editing safely and ethically.


Here to help

If you would like further information on patenting human genome editing inventions, contact Jamie Atkins or speak to your usual Kilburn & Strode advisor.

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