Abstract
Planting the Seed for an Editing Regulatory Framework in the European Union
2024 is off to a promising start with a resounding vote last month in support of new breeding technologies for plants by the European Union (EU). The European Parliament Committee on Environment, Public Health and Food Safety (ENVI) voted by a substantial majority (47–31) in favor of the proposed regulatory framework for new genomic techniques (NGTs). After years of preparation and informed policy consideration, the ENVI committee held a vote on the proposal entitled “Plants obtained by certain new genomic techniques and their food and feed,” amending Regulation 2017/625.
This is an important milestone—a game changer—for plant breeders in and beyond Europe, with a framework setting the stage for flexible editing in category 1 plants. (By contrast, category 2 transgenics remain subject to GMO regulation.) Critically, NGT category 1 plants will be considered equivalent to those obtained through conventional breeding and not require labeling. Technically, up to 20 genetic modifications will be allowed, encompassing substitutions or insertions up to 20 bases, deletions of any size, and sequences within the species gene pool. As this issue goes to press, we are anticipating the final assembly vote on February 6.
This momentous and encouraging recommendation is timely in light of expanded concerns about the global climate crisis and the need to more urgently harness disruptive technologies for more sustainable agriculture. There are implications for the scientific community, the agbiotech industry, and of course farmers and consumers, with the anticipated development of a more resilient and sustainable agriculture with benefits for our food supply, animal feed, and beyond. Presumably the considerable financial and bioeconomy impact was also taken into account.
This is important for the EU and its many trading partners in a contextually interconnected global food supply chain. This framework, together with precedents established elsewhere (e.g., USDA SECURE 7 C.F.R. Part 340), will provide a more globally relevant, convenient, and expeditious regulatory context to navigate for international players.
Besides the commendable resiliency of the ENVI committee and the many stakeholders involved in developing this regulation, it is noteworthy that leading scientists were compelled to reach out to members of the European Parliament just days before the vote with an open letter compelling them to make science-informed decisions and consider evidence-based arguments to assess NBTs. The letter, penned by nearly 40 Nobel Laureates (including our own CRISPR colleagues Jennifer Doudna and Emmanuelle Charpentier) and endorsed by >1,000 scientists, raised concerns about antiscience lobbyists and the associated fearmongering.
Whereas “controversial” aspects of genome editing are often invoked by these skeptical parties, it is important to highlight how scientifically consensual and united these scientific thought leaders are in their unequivocal support for CRISPR-based plant breeding technologies.
It is frustrating and concerning that there remains skepticism regarding the safety of CRISPR-based technologies, given the approval last year of safe and efficacious CRISPR medicines in the United States, the United Kingdom, and even the EU, and the authoritative reports (including the EU's own EFSA) about the safety of genome-edited crops and their commercialization in several countries, including the United Kingdom, post-Brexit. This is a stark reminder that politics still gets in the way of policies, and that the scientific community (and our readers) must continue to educate stakeholders, including regulators, politicians, and the public, to enable the timely deployment of genome editing.
Warrior Spirit: The Victoria Gray Story
We would rarely consider putting a patient on the cover of The CRISPR Journal, but recent developments fully justify the decision to celebrate the approval of Casgevy, a CRISPR-based cell therapy, for sickle cell disease (in December 2023) and beta-thalassemia (in January 2024).
In July 2019, Victoria Gray became the first U.S. sickle cell patient and pioneer to receive the experimental exa-cel therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The editing strategy targets the BCL11A repressor, resulting in a switch-up of fetal hemoglobin to compensate for the sickle cell point mutation in the beta globin gene. Victoria's swift and lasting recovery, and indeed that of the dozens of patients who followed her path, is inspiring not only to the many researchers who laid the path over the past 10–15 years developing CRISPR-Cas9 but also to the tens of thousands of sickle cell patients in the United States and millions more around the world.
Victoria is no stranger to giving interviews—she has been interviewed several times over the past 4 years by Rob Stein, a science reporter with National Public Radio, and gave a memorable talk at the Third International Summit on Human Genome Editing in London in March 2023. Last month, she agreed to be interviewed by our executive editor, Kevin Davies, and Genetic Engineering News’ reporter Uduak Thomas, as part of a virtual event, The State of Cell and Gene Therapy, broadcast on January 24. It was perhaps the longest interview she has given to date and one that we are thrilled to publish in this issue.
Victoria's remarkable and moving story, told in her own words (see page 5), illustrates the tangible benefits, safety, and efficacy of the first CRISPR medicines. Our hope is that it will also inspire other patients who might be contemplating this “one-and-done” therapy—assuming of course that they are able to afford it (Vertex Pharmaceuticals set the list price of Casgevy at $2.2 million).
Speaking of hemoglobinopathies, this issue also features a notable research article on prime editing from a team at the National Institutes of Health (NIH) led by Francis Collins, the former director of the NIH. Collins published his first article in Nature ∼40 years ago on the molecular genetics of hereditary persistence of fetal hemoglobin with Sherman Weissman and colleagues and retains a passionate interest in the clinical application of genome editing. In this issue, Lori Bonnycastle and colleagues used prime editing to generate a large series of isogenic induced pluripotent stem cell lines pertaining to type 2 diabetes, offering helpful guidelines to optimize editing efficiencies. We are delighted to be the platform to validate and disseminate this research.
All in all, a happy start to the new year.