FDA Approves Sickle Cell Drug that Uses CRISPR Gene Editing Tech

The FDA has approved gene-editing technology for the treatment of sickle cell disease. This is the same gene-editing technology expected to be used for various diseases, agriculture seeds, and synthetic, “cell-cultured” meat. The sickle cell treatment is called Casgevy and is being brought to market with partner Vertex Pharmaceuticals. Supporters and advocates have touted this technology as revolutionary, while detractors and studies have raised several concerns. 

A significant report came out in 2018 in China about a researcher who claimed to successfully genetically modify embryos that were eventually born as human babies. The modification was to make the childrens’ cells resistant to HIV infection despite the father being positive for HIV. This researcher spent three years in jail for an ethical and moral breach. CRISPR received funding in 2020 from the Bill and Melinda Gates Foundation to continue studying gene-editing treatments for HIV. Talk about designer babies and synthetic wombs has arisen as a result of this gene-editing technology. If the technology goes in that direction, it matches the definition of eugenics – “The study of how to arrange reproduction within a human population to increase the occurrence of heritable characteristics regarded as desirable.” 

Using CRISPR to treat sickle cell or any other disorder involves removing stem cells from a patient’s body. Then, a DNA sequence is deleted, or a new sequence is inserted. Then, the cells are placed back into the patient’s body. While this sounds like a form of genetically modifying DNA as agricultural giants Monsanto and others have done to create roundup-resistant seeds, authorities within the gene-editing industry insist this is a different kind of process that is more precise. 

Jeffrey Jaxen reported for The HighWire, “They can take pieces out, put them in, it recombines. That makes a whole new genomic species at that point. In old GMO foods, they were inserting genes; they were inserting herbicide-resistant genes and insect-resistant genes. We’re not inserting anything; we’re clipping and splicing.” Del Bigtree asserted that it is like surgery for DNA. 

Jaxen pointed to a study that stated, “Chromothripsis is extensive chromosome rearrangement restricted to one or a few chromosomes that can cause human congenital disease and cancer.” Jaxen explained that this is an “instantly cascading effect,” scientists, researchers, and geneticists do not know what happens downstream, but they can witness it happening in the lab.  

Jeffrey Jaxen’s report also covered the release of genetically engineered mosquitoes that were released in Florida by a biotech company called Oxitec, with EPA approval. While the intent of the GMO mosquitoes was to reduce the overall population and the risk of mosquito-borne illnesses, the first locally acquired malaria cases in the United States were reported this past June. 

A research paper published by Frontiers in Plant Science states, “Compared to traditional genetic engineering, where genetic modifications in the host genome were always random, CRISPR-Cas based modifications are precise, predictable, inheritable, and sometimes without introducing any external gene sequence in the host-genome.” 

This understanding of the CRISPR technology has caused industry leaders to change the label from “gene-editing” to “precision breeding” to bypass strict GMO regulations in the European Union. While the “precision breeding” bill was passed in the EU, it was not without pushback from gene therapy and plant sciences experts. In a letter with 103 signatories, Michael Antoniou breaks down his disagreement with using the words “precision” and “breeding” to describe this procedure. 

Antoniou explains, “Different types of unintended damage accumulate at the various stages of the gene editing procedure, at both the on-target site (the intended gene-editing site) and at off-target sites (elsewhere in the genome of the organism). A large number of peer-reviewed studies reveal unintended genetic changes from gene editing. A review of the literature shows that gene editing-induced changes are different from changes that occur in natural breeding, including mutagenesis breeding, because gene editing makes the whole genome accessible to changes, whereas in natural breeding, some regions of the genome are protected against mutations. Recent research in plants (not involving gene editing) confirmed that in natural breeding, mutations are not random and that certain regions of the genome involved in important processes are protected from mutations.”

The letter goes on to say that the gene-editing process bears no resemblance to breeding and encourages policymakers to change the marketing term of “precision breeding” to a more accurate term, such as “genetic modification technologies.” This is the same technology being developed to genetically edit the DNA of humans as a medical treatment option. One of the latest reports celebrates the success of lowering cholesterol in two out of ten study participants. 

This technology is expected to be studied for the treatment of many diseases, including Alzheimer’s, cancer, and AIDS. The statement from Antoniou signed by over 100 doctors, professors, and researchers, is alarming if research moves faster than regulatory bodies. Antoniou stated that a “large number of peer-reviewed studies reveal unintended genetic changes from gene editing.” Antoniou compiled a comprehensive literature review that can be accessed here. 

Politico reported on the new law in Brussels that makes it impossible for European countries to ban food that is bred with new genomic techniques (NGTs). The rules were pushed by Bayer, Syngenta, and Corteva, the main players in the NGT plant-breeding industry. 

The USDA has stated that “‘it does not currently regulate or have plans to regulate’ CRISPR edited crops. Under its biotechnology regulations, USDA does not currently regulate or have plans to regulate plants that could otherwise have been developed through traditional breeding techniques as long as they are developed without the use of a plant pest as the donor or vector, and they are not themselves plant pests.”

The USDA and EPA have deregulated CRISPR and gene-edited plants in the case that they could theoretically be created through natural breeding. Donald Trump signed an executive order in 2019 calling for low-risk genetically engineered plants to be deregulated. Joe Biden signed an executive order in 2022 declaring, “We need to develop genetic engineering technologies and techniques to be able to write circuitry for cells and predictably program biology in the same way in which we write software and program computers; unlock the power of biological data, including through computing tools and artificial intelligence.”