Background

CRISPR-Cas9 genome editing has been a topical subject since the Chinese research group led by Junjiu Huang at Sun Yat-sen University in Guangzhou used this technology to eliminate the human β-globulin (HBB) gene from the germline of a non-viable human embryo. Naturally occurring mutations in HBB cause the blood disorder β-thalassaemia, which can be fatal. The group's research article was rejected by both Nature and Science on ethical grounds and was eventually published in the journal Protein and Cell. Subsequently, in a groundbreaking decision, regulatory approval for a research study using CRISPR-Cas9 genome editing in viable human embryo cells was provided in the United Kingdom by the Human Fertilisation and Embryology Authority to Kathy Niakan's team at the Francis Crick Institute.

Despite the obvious potential benefits of this technology for the development of therapeutic treatment of otherwise incurable genetic and other diseases, ethical concerns over its therapeutic use are being debated around the globe. The biggest, but not the only, concerns over the use of this technology as a therapy are the possible off-target mutations that might arise during the use of the editing method, which would then be passed on to the patient's progeny.

In South Africa, gene editing techniques have and are being used in therapeutic research studies with adult human cells. However, the ethical concerns around somatic gene editing therapy are less controversial than with germline therapy. Any possible adverse effects in somatic cells would affect only the patient in question, and the treatment's risks can be assessed and weighed against the potential benefits. Conversely, germline mutations are transferred to all progeny cells and would be passed on to subsequent generations of children and form part of the human gene pool.

Patentability considerations

A number of patent applications relating to the use of CRISPR-Cas9 technology have been filed in South Africa by the Massachusetts Institute of Technology, the Broad Institute and the President and Fellows of Harvard College, among others. None have yet proceeded to grant.

The Patents Act (57/1978) does not preclude patenting of germline editing technology for human gametes or the products of such methods. However, certain inventions of this type may fall within the provisions of the Patents Act relating to offensive or immoral behaviour and may therefore be considered to be unpatentable. Since there is no substantive examination of patent applications in South Africa at present, it is questionable whether the registrar will ever refuse an application on such a ground. Another possible course of action for a third party wanting to challenge such a patent would be for it to apply for revocation of the patent once granted. One of the Patent Act's grounds for revocation of a granted patent is that it should not have been granted as it pertains to unpatentable subject matter. These provisions all hinge on a question of morality and whether the invention would generally be considered immoral by either the registrar or the courts, and such a consideration has yet to be made.

Legislative considerations

Obtaining a granted patent is not a guarantee that the patented technology may be freely used. The National Health Act (61/2004) and its regulations – in particular, the Regulations relating to Control of Use of Blood, Blood Products, Tissue and Gametes in Humans, which were promulgated in 2012 – regulate the removal and use of tissue, blood, blood products and gametes from living persons. The use of such materials in techniques involving germline editing, although not specifically referenced, would also be regulated by the National Health Act.

At present, removal of any such materials, apart from gametes, requires written consent from the subject if he or she is over the age of 18 or from the subject's parent or guardian if he or she is under the age of 18. In the case of gametes, such material may be removed only if the subject is over the age of 18 or ministerial approval is obtained. Additionally, the National Health Act places restrictions on the reproductive cloning of human beings which would be part of the process where germline editing is used on a viable human embryo.

The National Health Act prohibits the manipulation of genetic material of human gametes, zygotes or embryos and activities such as nuclear transfer or embryo splitting for the purpose of the reproductive cloning of a human being. The minister of health may permit research on stem cells and zygotes which are younger than 14 days old, on a written application for research purposes, so long as the applicant undertakes to document the research and the donor's prior written consent is obtained.

Any person who fails to comply with the National Health Act regulations will be guilty of an offence and liable on conviction to a fine, 10 years' imprisonment or both.

Comment

Members of the scientific community have argued for a moratorium to be called on human germline editing and the US National Institute of Health (NIH) has banned NIH-funded research into the genomic editing of human embryos. However, other members of the scientific community have argued that it is unethical to withhold technology that might be used to treat otherwise incurable diseases.

At present, the modification of a human embryo's germline for therapeutic purposes culminating in the reproduction of a human being is prohibited in South Africa. Germline editing for research purposes might be permitted, but would require conditional ministerial approval.

For further information on this topic please contact Joanne van Harmelen at ENSafrica by telephone (+27 21 410 2500) or email (jvanharmelen@ensafrica.com). The ENSafrica website can be accessed at www.ensafrica.com.

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