Three research and industry institutions Nov. 14 testified before the Senate Health, Education, Labor, and Pensions (HELP) Committee about the role of gene editing technology, particularly CRISPR/Cas9, in developing treatments for genetic disorders as well as other uses. The hearing’s goal was to understand the implications of these advancements and ensure that the National Institutes of Health (NIH) and other federal programs are able to support this research safely and effectively. Committee members of both parties expressed bipartisan support of gene editing technology and its potential to solve many health problems, as well as current regulations of these technologies, recently updated in the 21st Century Cures Act of 2016.
In her opening statement, Ranking Member Patty Murray (D-Wash.) espoused the promise of these new technologies, “Gene editing technology has the potential to be used as a tool to tackle difficult research questions, treatment for serious genetic diseases, ... an approach to engineering our own cells to fight cancer and infections, and a new way to help stop the spread of infections.”
While they discussed several regulatory concerns, the witnesses agreed that current regulations were appropriate and that they created a robust research environment. The witnesses expressed a need for some flexibility, so that as more data is gathered, regulations adapt and remain data-driven. Committee members emphasized the importance of genetic data privacy in light of the need for data sharing to advance both basic and industrial research, and members agreed that privacy was well protected in the 21st Century Cures Act. While witnesses stated that gene editing technology has not yet advanced enough to be used in hereditary human germline editing, current thinking, also expressed in a recent National Academies of Science report, is that potential future uses of gene editing technology in this context must be strictly regulated - used only for great need and when no other treatments are available.
Conversation about gene editing technology on the global stage centered on ensuring the United States remains a leader in the field. Witnesses were concerned that if the technology was prohibited in the U.S., the result would be a loss of American and international scientists to countries with fewer or no restrictions. Thus, the U.S. would not only lose any benefits from the technology, it also would lose any control of the direction of research. Witnesses also stressed that the U.S. must remain a leader in biodefense against harmful uses of gene editing technology. When asked about how the U.S. could help ensure ethical use abroad, witnesses described the ongoing international dialogue with the U.K., China, and other countries.
Also noted throughout the hearing was the importance of government funding of basic research in discovering CRISPR. As Chairperson Lamar Alexander (R-Tenn.) stated, “CRISPR is just one of the amazing discoveries that has come from basic research, funded in part by the federal government.”
Witnesses included Matthew Porteus, PhD, MD, Associate Professor Of Pediatrics, Stanford University; Katrine Bosley, CEO And President, Editas Medicine; and Jeffrey Kahn, PhD, MD, Director, Johns Hopkins Berman Institute of Bioethics.