Principles for genome editing developed

Out-Law News | 29 Aug 2019 | 8:31 am | 2 min. read

Gene editing technology is not yet ready to be used to pass genetic changes down to future generations, according to a number of biotech companies.

Thirteen businesses that use gene editing technologies confirmed their view on the topic as they endorsed a new "bioethical framework for the use of gene editing in therapeutic applications". The statement of principles was developed under the umbrella of international lobby group The Alliance for Regenerative Medicine.

"Unless and until ethical and potential safety questions with respect to germline gene editing are adequately addressed, we do not support or condone germline gene editing in human clinical trials or for human implantation," the statement said. "We believe that these are international concerns and would be supportive of an effort to discuss therapeutic gene editing issues on a global stage."

The companies said in the statement that they do support efforts to find new ways to use gene editing, but only in the context of somatic cells. Those are cells unique to individuals and are different to germ line cells which can be passed down to future generations.

"We do not support gene editing of the germline (sperm, eggs, fertilized embryos) for the purposes of human implantation," the statement said. "Further, we do not support implantation of a human embryo carrying gene modified cells. Most genetic diseases can be treated directly in affected somatic cells without modifying the germline, and we support such therapeutic use."

"Regulated, clinical validation of somatic cell-based gene editing technologies for non-inherited genetic modification is, and should remain, the primary objective of the therapeutic development community and, in contrast to germline gene editing, offers the most acceptable near-term path to potentially transformative therapeutic benefits for patients," it said.

"Gene editing technologies have not matured to the point where human trials of edited germline cells are appropriate. Many important safety, ethical, legal, and societal issues involved with this type of gene editing remain unresolved," it said.

The signatories also gave their support to efforts to develop new gene editing standards, and further called for an evolution in regulation in the market.

"We believe that evolving national and regional regulatory frameworks are important to support appropriate development of these technologies and should act as the primary regulatory and enforcement mechanism," the statement said. "It is our belief that arbitrary and ancillary oversight bodies or processes may carry the risk of delaying research and development efforts, which in turn would adversely impact afflicted patient populations."

Gene editing has been the subject of controversy in recent times. Chinese state media reported earlier this year that an investigation in the country had found that a scientist who claimed to have used gene-editing technology to alter the DNA of two new babies in China had done so illegally.

Life sciences expert Helen Cline of Pinsent Masons, the law firm behind Out-Law, said: "The issue being addressed is the need for oversight at a global level, not the fundamental benefits of genome editing. It is the scientists themselves that are considering how best to regulate gene editing techniques such as CRISPR and calling for guidelines so that research in this area can proceed in a responsible way, with the right oversight and quality controls."

"The European Medicines Agency (EMA) in Europe and the US Food and Drug Administration (FDA) already oversee the development and commercialisation of promising new somatic gene therapies. However, even here ethical questions remain, including where to draw the line between disease treatment and enhancement," she said.