As an industry that, at its root, exists to improve health and meet patient needs, we have a responsibility to engage with patients and society to develop the best possible treatment pathways for them. This is a hot topic in medicines development, from increasing patient involvement in research design, to the use of health data to evaluate treatment. But what about the techniques and methods we use to design medicines themselves? Does the way in which new therapies work raise concerns for patients? Do our scientists have an obligation to engage with and listen to society’s views?
The Nuffield Council of Bioethics has today launched their review on genome editing, highlighting the diversity of challenges, views, and perspectives on this technology across science and society, and areas for focus going forwards.
Genome editing is the alteration of the DNA sequence in a cell, which can go on to impact the production of proteins and cellular machinery. Techniques developed within the last 15 years have made editing much more precise, allowing scientists to make specific changes to DNA sequences. The most recently developed of these techniques, the CRISPR-Cas 9 system, has hugely decreased the cost and difficulty of genome editing. As a result, use of the technique has spread rapidly through the scientific community, and it is now used widely across the life science sector and beyond. This rapid diffusion is one of the main concerns highlighted throughout the review, as advances in science and its application may outstrip the pace at which society can reflect, develop, and implement appropriate governance and norms for its use.
Within the biopharmaceutical industry, genome editing has two main applications: biomedical research into disease and new treatments, and potential use in gene and cell therapies. For research, genome editing allows the rapid creation of cell and animal models of disease. This opens the possibility of 'personalised models' of cells for individual patients, which should accelerate the discovery and testing of new medicines. But genome editing is also generating significant interest as a potential treatment in its own right. A number of biotech companies has emerged and attracted significant investment into this area. Companies such as Caribou Biosciences, Editas Medicine, and Intellia Therapeutics are actively exploring the use of genome editing techniques to treat disease, primarily rare hereditary disorders or in immune cells to target cancer, and in some cases are near entering the clinic.
These applications have the potential to bring significant benefits to human health. They are also much less controversial and ethically problematic than some other areas where genome editing could be applied, such as editing of human embryos to avoid disease or for enhancement, or editing livestock to enhance food production. However, even in the relatively safe realm in which the biopharmaceutical industry operates with respect to genome editing, the use of such techniques raises a myriad of ethical questions. Those highlighted in the Nuffield Review include:
None of these challenges should put a halt on the use of genome editing techniques in medical research. However, they highlight the importance of further research into the techniques, the necessity of continued development of robust governance and standards for the field, and responsibility for scientific engagement with the diverse views in democratic society. Maybe, therefore, this topic is a new opportunity for industry to move patient and public engagement back up the development pathway, not just to clinical research, but to science and drug discovery itself?