Questioning the direction of genome editing
Posted by University College London on 06 Jul 2015
From the public understanding of science movement in the 1980s and early 1990s, through public engagement with science and most recently responsible research and innovation, science is gradually opening up to wider public involvement. Part of the point of engagement is to explore openly the directions, purposes and motivations behind research before aims and research trajectories become immovable.
As new technologies snowball towards markets promising newer, better and faster but threatening harmful, irreversible and disruptive downsides, their direction of travel should be watched carefully.
Drawing on lessons from the controversy over genetically modified organisms at the turn of the century, European policy dealing with synthetic biology makes promising gestures towards fostering an open and upstream dialogue on research into the technology. This approach reflects a commitment among parts of the synthetic biology community to keep the research itself, and its tools, open-source.
Contrast the approach taken with genome editing, currently attracting specialist attention due to the recent discovery of a faster, cheaper technique called CRISPR/Cas9. CRISPR/Cas9 retains the principle of earlier genome editing techniques – ZFNs and TALENs – which involve cutting an organism's DNA to enable a desired change, for example removing unwanted genes or inserting new genes. However, instead of using proteins, which are slow and expensive to manufacture, to identify the location to be cut, CRSIPR/Cas9 uses RNA molecules, which are quicker and cheaper to manufacture. Patenting and venture capital funding swiftly arrived, anticipating the leap forward in genome editing this development would trigger, and the commercial start-up, Editas Medicine, was established to exploit both CRISPR/Cas9 and TALENs. Genome editing could lead to gene therapies for diseases like sickle-cell anaemia and cystic fibrosis; or in agriculture, research into plant gene function or development of new traits. However, these promises come with attendant risks and difficulties. Off-target mutations, which could cause harm, are possible. Furthermore, the speed of R&D in this area currently outpaces ethical and regulatory oversight. In contrast to synthetic biology perhaps, research into the technology, at least in Europe, has so far continued out of the public spotlight. This may be partly due to the fact that it has not yet fully pierced the regulator's consciousness. There is also currently a race to assert property rights over the technique, described in Nature as the "CRISPR IP land grab".
Another emerging biotechnology raising questions of openness and ownership is that of engineered gene drives, now more achievable since the advent of CRISPR/Cas9. "Gene drive" describes a phenomenon in nature which involves genes increasing their chances of being inherited and thereby becoming widely dispersed throughout a population. Engineered gene drive entails harnessing this phenomenon by using biotechnology to design and produce desired gene drives, which can be used for example, to tackle agricultural pests and the spread of infectious diseases such as malaria. Recognising the risk of drastic, irreversible and unpredictable impacts on both human societies and entire ecosystems, such as unintentional spreading into closely related species through unusual mating, pioneers in this field have called for broad public discussion focusing on their responsible use. The call is welcome. However, a closer look reveals patents have already been granted on RNA-guided gene drives.
In the absence of formal regulations, the application of Intellectual Property rights to new technologies or components imposes its own system of governance on the technology. At the laboratory level, the "land grab" means that researchers in genome editing will either have to avoid using the patented material in their own research or pay for cross-licences, which could hinder research. Patents are part of modern science. However, the achievements in opening up science to broader engagement need to extend to reflecting not only on its directions, purposes and motivations but also on the implications the presence of patents have for all of these. Depending on its implementation, patent law can provide space to air (moral) concerns. It can also restrict debate, or at least the terms of debate. This is not to say that private ownership necessarily forecloses public benefit, but such moves demand wider scrutiny.
This blog post draws on a briefing note published here.
Olivia Hamlyn is a PhD researcher at UCL, and a member of the UCL Hub for Responsible Research and Innovation