A consensus study of the scientific underpinnings of human gene editing technologies is underway, and the committee of experts behind the effort will independently review potential applications for those technologies, as well as the clinical, ethical, legal, and social implications of their use.
The multidisciplinary committee met Feb. 11 to receive input from select stakeholders as it launches its review, the results of which will represent the official views of the National Academy of Sciences and the National Academy of Medicine.
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Among those stakeholders were representatives from several companies commercializing gene editing, a representative from the National Institutes of Health, and patient advocacy groups.
With some caveats, participants lobbied to maintain the existing regulatory framework related to gene therapy.
Michael Werner, cofounder and executive director of the Alliance for Regenerative Medicine, which advocates globally for regenerative medicine and advanced therapies, said his organization believes “the existing regulatory framework overall works for these technologies.”
“We don’t believe the [Food and Drug Administration], for example, needs to create a whole separate oversight process in addition to the process we already have now for gene therapy,” he said, adding that “the ultimate goal here is not to have regulatory action or legislative action that will hinder or delay the development of these technologies.”
The consensus study follows an International Summit on Human Gene Editing held in December, and is the next component of the Human Gene Editing Initiative. The committee will study the potential for gene editing in biomedical research and medicine – including human germline editing – although representatives from each of the companies represented at the meeting noted that they are not currently focused on germline applications.
Rather, the commercial focus is on other areas, such as correcting genes in somatic cells.
“We start with medical need. We want to work on things where there is not currently a therapy that addresses adequately the medical need, and we need the ability to generate a differentiated product,” said Vic Myer, Ph.D., of Editas Medicine in Cambridge, Mass.
The company has projects underway for opthalmologic applications, sickle cell anemia, beta-thalassemia, Duchenne muscular dystrophy, cystic fibrosis, alpha1-antitrypsin deficiency in the liver, and others.
“We are working on a number of different types of edits in a number of different tissues with a number of delivery modalities,” Dr. Myer said, noting that some programs will move quickly, while others will not.
Similarly, Intellia Therapeutics, also of Cambridge, Mass., is “focusing very much on what we hope will be curative products for somatic gene-based disorders,” said Dr. John Leonard, the company’s chief medical officer.
“We limit our work to somatic cells. We thought very carefully about that and that is what we do,” he added, noting that a recently launched division of Intellia is focused on autoimmune oncology opportunities (ex vivo), and on liver disease (in vivo).
The committee of experts conducting the consensus study, which began its information-gathering process in December at the International Summit on Human Gene Editing, will take these and other views expressed at the meeting into consideration during its work over the next year. The study will include a literature review and data gathering via meetings in the United States and abroad. The committee will continue to seek input from researchers, clinicians, policy makers, and the public.
“The committee will also monitor in real time the latest scientific achievements of importance in this rapidly developing field,” according to information from the National Academies.
The field has enormous potential, according to Mr. Werner, who explained that combined, the various types of gene editing technology available are “pretty powerful” in terms of an approach for targeting and changing DNA sequences in human cells.
“We’re talking about the potential to durably treat and potentially even cure diseases that currently represent unmet medical needs,” he said. “We could, in theory, be talking about millions of patients worldwide.”