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There's a fine red line between cures, enhancements using gene editing tech


By Nuala Moran
Staff Writer

BOSTON – The triumph of the Massachusetts Institute of Technology Broad Institute and its spinout company Editas Medicine Inc. in the case of CRISPR/Cas9 gene editing patents could impede the development of human therapies based on the technology, according to one of the leading researchers in the field.

George Church, professor of genetics at Harvard Medical School, said, "It definitely is an issue" if Editas maintains exclusivity in applying patents on using CRISPR/Cas9 to edit eukaryote genes. That would limit the freedom to operate of Editas' direct competitors, Intellia Therapeutics Inc. and Crispr Therapeutics AG.

Church added that even if all three companies are equally involved in translation, "that is not enough to handle all the benefits to come out of this. I would love to see more companies," he told AAAS attendees.

Church could be seen as having something of an axe to grind because he published a paper in the same issue of Science as Feng Zhang of the Broad Institute, on using CRISPR/Cas9 in human cells. It is that research of Zhang's on which the disputed patent hangs.

The decision on the high-profile CRISPR/Cas9 patents came a day after the Committee on Human Gene Editing of the National Academies of Sciences and Medicine issued a report concluding that clinical trials involving genome editing in gametes or early embryos could be permitted in the future for serious diseases or disabilities, under stringent oversight. (See BioWorld Today, Feb. 15, 2017, and Feb. 16, 2017.)

Church was one of the contributors to the international summit on gene editing held in Washington in December 2015 that led to the writing of the report. He said the report's proposals amounted to "a change in the red lines we are drawing." When talking about altering the inherited germline and the somatic cells of adults, "the line is now drawn on the seriousness of the disease," he said.

While the red line in germline modification is shifted, the line between using gene editing to cure disease and to enhance human traits becomes softer, Church suggested. For example, gene editing somatic cells to increase muscular strength in patients with muscular dystrophy could tip over into giving subjects greater than average strength.

Similarly, a somatic gene editing to improve cognition as a "cure" for Alzheimer's disease could result in patients having enhanced cognitive abilities.

Such modifications are far from becoming reality, but Church said other traits are more amenable to change. One example would be somatic modification to promote endogenous production of human growth hormone, which could be viewed both as a therapy and as a means of enhancement.

The Committee on Human Gene Editing was convened to try to get ahead of the curve in establishing regulations, before those types of modifications become feasible, as Richard Hynes, professor of cancer at MIT and co-chair of the committee, explained.

While he agreed it is difficult to draw the line between the use of somatic gene editing for treatment of disease and for enhancement, he said he firmly believes enhancement should be banned for now. "We should work out the risk/benefit for therapies first. It will take time to understand the risks. With enhancement, the benefits are debatable, but the risks are the same," Hynes said.

The concern is that, as with stem cell therapies, genetic enhancement through somatic gene editing will be on offer in rogue clinics and in countries where there is limited oversight.

Given that, one of the aims of the committee was to set out core principles that would be broadly influential and could be adopted as the basis for promulgating national laws governing the technology. "The principles are for application around the world, as part of a drive to try and harmonize international regulation," Hynes said.

That is all very well, but as Gary Marchant, of Arizona State University, also a member of the committee, noted, "There is a huge problem of international enforcement." Ways of trying to ensure compliance considered in the report include journals only publishing research that complies with international norms and withholding research grants if the rules are not upheld.

The committee did not consider patenting in its survey of gene editing. However, Marchant suggested refusing licenses could be another mechanism in attempts to ensure appropriate use of the technology worldwide. That "may have more currency at an international level," he said.