In 1997, the United Nations Educational, Scientific and Cultural Organization (UNESCO) declared the human genome the “heritage of humanity.” This symbolic title recognized the importance of protecting the human genome as something to be dutifully passed on to future generations. When this declaration was signed, the sequence of the first human genome had yet to be published and genome editing tools were rudimentary. Twenty years later, the recognition of the human genome as something worth guarding has taken on a profound sense of urgency. The astonishing rise of sophisticated genome editing technologies is turning hypotheticals into inevitabilities, a reality that Kevin Davies explores in his latest book Editing Humanity.
The title of this book captures what UNESCO asserted twenty-five years prior—that by heritably altering even one human genome, the entirety of humanity is impacted through its shared heritage. This nuance becomes even more apparent at the book’s apex, the gripping story of He Jiankui (known as JK), a Chinese scientist who is now immortalized in history books as the first person to allegedly create genome-edited babies, opening a Pandora’s box that many people argued can no longer be closed.
The release of Davies’s Editing Humanity could not have been better timed. One day after publication, the 2020 Nobel Prize for Chemistry was awarded to Jennifer Doudna and Emmanuelle Charpentier “for the development of a method for genome editing,” only eight years after their initial discovery. Davies’s book is one among many that have sought to capture the arc of the CRISPR story. The Code Breaker by Walter Isaacson was published not long after Davies’s, and even the scientists at the heart of these stories have documented their groundbreaking discoveries (e.g., A Crack in Creation by Jennifer Doudna) and described their eccentric applications (e.g., Regenesis by George Church). What sets Davies’s book apart is his ability to expose the CRISPR story and its nuances from numerous, sometimes conflicting, perspectives. While the center of the story is a billion-year-old bacterial immune system, what keeps the reader engaged is the cast of characters transforming that natural system into a multi-billion-dollar industry.
This is not Davies’s first attempt at unraveling a convoluted and contentious scientific odyssey filled with peculiar researchers. In Cracking the Genome, he follows the Human Genome Project (HGP) and the drama that unfolded between public and private researchers racing towards sequencing the first draft of the human genome. As founding editor of the leading journal, Nature Genetics, Davies had the scientific proficiency and backdoor access to HGP’s pivotal players. In Editing Humanity, he takes a similar intimate approach, enjoying a drink with distinguished scientists, sitting front row at exclusive conferences, and sharing jokes with Nobel laureates.
While Davies jumps between years, stories, and characters, his book follows a logical arc. This review attempts to mirror that thematic trajectory by focusing on four of its sections. First, this review discusses how Davies introduces main and auxiliary characters, stressing the critical role of credit and priority in shaping how scientists are viewed and treated. Second, this review juxtaposes the popularized simplicity and feasibility of CRISPR against the technological and legal factors that either limit or constrain certain applications. Third, underscoring the author’s journalistic ability to capture the nuance of a controversial figure, this review examines the unique portrayal of JK within broader societal and political contexts. Fourth, this review critiques Davies’s choice of genome editing applications, arguing that the author takes a techno-optimistic view of these use cases that is made evident by his characterization of scientists and description of their research. The review ends with select updates on governance, clinical uses, and global equity that have impacted the field since the book’s publication.
The story of CRISPR can be condensed into a simple eureka moment, an abrupt stroke of genius. To some extent, this narrative is true. A brainstorming session between Jennifer Doudna and her postdoc Martin Jinek led to the realization that the Cas9 protein could be programmed using a synthetic guide RNA to target and cut any sequence of DNA. But in many respects this characterization of the CRISPR story is woefully incomplete and an unfortunate portrayal of the more accurate and serendipitous story, one which Davies embarks to depict.
Davies takes time to introduce a broad cast of scientific characters that have played a broad spectrum of roles in CRISPR’s transition from a bacterial immune system to a genome editing tool and beyond. This holistic picture encompasses parallel instances of discovery and competing claims of invention, creating a contentious story that he methodically unravels. Davies makes sure the reader knows the exact order of events, making clear that in science, order matters.
As Leonid Tiokhin and his coauthors have observed, “The priority rule is a particularly longstanding scientific norm, in which individuals who are first to make discoveries receive disproportionate credit relative to all other individuals who provide solutions to the same problem.” Being first comes with a myriad of scientific, social, and entrepreneurial benefits, yet this important distinction is not bestowed by a single entity. Instead, the public, media, peers, and legal systems, all contribute to labeling a discovery or invention as a scientific first—sometimes in conflicting ways.
Within the first few chapters, Davies introduces a rolodex of scientists who have attracted notoriety and have been given credit for consequential CRISPR-related discoveries. Beginning in the salt flats of Spain, Francisco Mojica identified CRISPR sequences in the DNA of single-celled microorganisms, leading to what he says was the “first paper where CRISPR was taken seriously,” published in 2004. Three years later, a team from the Danish yogurt company Danisco, including Philippe Horvath and Rodolphe Barrangou, published their results demonstrating the role of CRISPR in defending bacteria from viral infection.
Fast forward to 2012, and scientists from around the world were racing to repurpose CRISPR proteins into RNA-guided tools for targeted DNA cutting. The eventual Nobel prize-winning team of Jennifer Doudna and Emmanuelle Charpentier published their findings first in June 2012 despite a team, led by Virginijus Šikšnys, from Lithuania who submitted their manuscript for review in March 2012. Arguably the most significant instance of scientific priority occurred in January 2013, when Feng Zhang and George Church demonstrated CRISPR editing in human cells, showing that human genome editing using CRISPR was possible.
Through these stories, Davies dives deep into the politics of science, illustrating the enormous implications of receiving priority. In the end, the scientific publishing enterprise gave priority to Doudna and Charpentier over Šikšnys. As for Zhang, the legal system gave him priority for the development of CRISPR as a genome editing tool, resulting in ownership of intellectual property that would be worth billions of dollars.
Along the way, Davies makes a noticeable effort to mention the valuable contributions of collaborators, postdocs, and students. These essential role-players are too often left out of the history books, yet every instance of CRISPR discovery and invention was the result of two or more minds, and never a lone scientist in an ivory tower. As Davies emphasizes, “Most scientific papers are team efforts, the fruit of months if not years of planning, reviewing, and repeating experiments, a continual exchange of ideas between student and mentor.” The fruits of such labor are rarely evident until years or decades later.
CRISPR is the perfect example of delayed impact, demonstrating the importance of basic and exploratory research and the value of pursuing questions without clear applications. The scientists examining bacterial immune systems were not looking for the next big biotech tool. Instead they were following the breadcrumbs of nature, chasing clues that eventually revealed a billion-year-old arms race. Editing Humanity can be used as an argument for increasing federal funding for basic research and supporting international collaborations.
CRISPR is heralded as a cheap, fast, and effective tool that is quickly democratizing genome editing. To some degree, this description is accurate, particularly compared to earlier technologies, which were costly and cumbersome. But in many ways, this characterization is an oversimplification, misrepresenting CRISPR’s limitations and trivializing the long road ahead towards disrupting modern medicine and agriculture. While Davies introduces several critical hurdles restricting CRISPR’s immediate impact, three are worth highlighting here: editing efficacy, cellular delivery, and biological understanding.
To forecast the future capabilities of genome editing, we must understand the evolution of the field. Davies takes the reader through the history of genome editing, sometimes referred to as genome editing B.C. (before CRISPR). Focusing on zinc-finger nucleases (ZFNs), Davies draws a line from the first experiments attempting intentional genetic manipulation in living cells through to the first clinical testing of genome editing technologies. These tools were either error prone or expensive to create, making CRISPR a monumental step forward owing to its relatively easy programmability and low cost. Near the end of the book, Davies notes improvements made to first-generation CRISPR technologies and the discovery of new DNA-cutting proteins, bringing scientists a few steps closer to the ultimate goal of making any genetic change, at any location, in any organism. But scientists are still years away from this utopian vision of genome editing.
Questions still loom about what exactly happens inside cells once these molecular machines are added. Davies references concerns regarding off-target effects, namely how efficient CRISPR proteins are in targeting specific regions of the genome without disrupting other DNA sequences. But even achieving 100 percent efficacy is not without its concerns. On-target effects, referring to unintended genetic rearrangements that occur at the site of an intended DNA cut, remain an unresolved issue. Uncertainty around off-target, on-target, and other unintended genetic alterations is part of the reason why the U.S. Food and Drug Administration (FDA) recommends patients be followed for fifteen years following any CRISPR procedure, making sure doctors can monitor any unexpected adverse outcomes.
The FDA is not only concerned with what happens inside a genetically engineered cell; the agency also closely monitors what happens outside the cell. In particular, the delivery of genome editing machinery into single cells or whole organisms introduces many technical and safety challenges. These challenges have been evident since the early days of gene therapy and the death of Jesse Gelsinger. Jesse was an eighteen-year-old with a rare genetic disorder called ornithine transcarbamylase (OTC), a disease that prevented him from processing nitrogen thus causing a build-up of toxic ammonia. In 1999, Jesse received an injection of viruses carrying a healthy copy of the OTC gene. His body’s deadly reaction to the viruses would make Jesse the first-known person to die in a clinical trial for gene therapy.
Problems with viral delivery vehicles and other means of introducing DNA into specified human cells would remain a troublesome bottleneck for the gene therapy field for decades. Scientists are experimenting with new ways to target specific cells across the body, particularly cells that cannot be removed and reintroduced like brain or lung cells. Overcoming these challenges will be essential in addressing diseases like Huntington’s disease and cystic fibrosis.
Even if scientists engineer effective genome editing tools and develop safe cellular delivery vehicles, a critical, and often underappreciated, rate-limiting step to treating debilitating diseases or engineering crops comes down to basic biological understanding. Davies makes this point by describing the circuitous journey towards a cure for sickle cell disease. Sickle cell disease is considered low-hanging fruit when it comes to genome editing. Everyone who has this disease has the same single-letter mutation in the human subunit beta (HBB) gene. Scientists know what cells to edit to permanently address this disease. They even know how many cells to edit. Yet one of the most effective ways to cure this disease using CRISPR involves a completely different gene than the one causing the disease.
By altering DNA within the BCL11A gene, scientists can increase the amount of fetal hemoglobin produced in a patient’s body, alleviating symptoms associated with sickle cell disease. This realization came decades after the genetic cause of sickle cell disease was discovered and forms the basis for multiple gene therapy clinical trials. This story highlights how the most effective way to treat a disease using CRISPR may involve targeting DNA sequences thousands or millions of letters away from the mutated gene. For many diseases, this level of biological understanding is unavailable, acting as a bottleneck limiting the clinical impact of genome editing.
In many ways this section feels like the climax of the book. In earlier chapters, Davies describes the capabilities of genome editing, while offering probing questions of how far those capabilities can be pushed and hurdles holding back certain applications. He notes: “What if we take our molecular scissors and repair some of the more than 75,000 mutations, deletions, and rearrangements that give rise to genetic diseases? What if, in the words of Chris Martin in fact, we could “‘fix you’—genetically speaking?” The logical next question becomes, why not make these changes at the single-cell stage before a baby is born?
While most of the scientists that Davies interviewed accepted the inevitability of germline genome editing, most were shocked by the circumstances of the first attempt from how the news was announced to the chosen genetic change to even the scientists leading the effort. Everything about JK’s story was unanticipated, making for a gripping tale that Davies walks the reader through in meticulous detail.
The story of JK mirrors a Hollywood script—a rogue scientist seeking international acclaim who flew too close to the sun of academic immortality. With a story as polarizing as JK’s quest for fame, Davies had the option of picking a side, making him the hero or the villain. Instead, Davies chose to toe the line, ultimately leaving the reader to come to their own conclusions about this once obscure and now infamous scientist.
Davies pulls off this feat of literary acrobatics by taking the reader behind the scenes, into secretive meetings, outlining who knew what and when. The initial sentiment is one of repulsion. Davies shares the reaction of a scientist who was one of the first to hear the news: “Musunuru said his heart sank as he opened the file. He called it ‘a soul-destroying moment.’ This wasn’t a hoax. If anything, it was worse.” But as the reader learns more about the scientist behind the experiment, disdain turns into understanding with maybe a hint of empathy.
Davies provides a thorough analysis of JK’s flawed attempt of germline editing. He describes the sloppy experiments and unethical obtainment of improper patient consent, but choses to end this section of the book questioning the common take on JK’s story, stating “It’s a convenient narrative, but is this the full story? I have my doubts.”
First, JK wasn’t a little-known scientist. He was a rising star in the Chinese scientific community, described as the “new top shot in the gene world.” Second, there is reason to believe that the Chinese government knew what JK was doing and may have assisted directly or indirectly. Third, China was motivated to forge ahead with clinical applications of CRISPR technology, having already treated almost one hundred patients for various cancers by 2017. Fourth, bioethics is a relatively young scientific discipline in China with less government enforcement compared to other countries deploying genome editing. Fifth, the one child policy of China had created a culture of fertility control and optimization.
All of these factors and more created an environment that would contextually justify JK’s actions. Davies declares that “JK’s actions were China’s responsibility” and although members of the scientific community across the world knew his intentions, they chose to do nothing. In his nuanced approach to covering the CRISPR baby scandal, Davies showcases his journalistic experience, presenting all sides of the story and placing the actions of a few people within a broader global context.
Headlines describe CRISPR as a “revolutionary” technology—an incredible discovery igniting a genome editing “revolution.” While this is accurate in some regards, considering its impact on scientific research (e.g., drug discovery), such a description is largely premature. Revolutions are defined as a dramatic change; the overthrowing of a system for a new way of doing things. Revolutions are retrospective, looking back and seeing a clear demarcation between how things were and where they are now. In the book’s final section, Davies explores areas where CRISPR could instigate profound revolutions, but sometimes the reader is left to wonder whether it is the right revolution or whether a revolution is required at all.
As of June 2021, nearly 3,300 species of animals have had their genomes sequenced and made publicly available. This abundance of data is offering novel insight into species evolution and environmental adaptation. Yet, beyond serving as a commodity for basic scientific inquiry, synthetic biologists are using this information to alter animal genomes as means to some great ends, applying the latest genome editing tools with a level of creativity only matched by its boldness. One scientist quick to push the limits of animal editing is George Church at Harvard University. His use of genome editing could be critiqued as “a solution in search of a problem,” but nonetheless his scientific footprint is being left across the animal kingdom. Earlier in the book, Davies detailed Church’s foundational role in demonstrating the feasibility of CRISPR genome editing in human cells. Now, Davies pivots to a cast of scientists spawned from the Church lab with entrepreneurial aspirations and a provocation for the unconventional.
Davies first unveils the idea of xenotransplantation, a small yet emerging field involving the transplantation of living cells, tissues, or organs from one species to another. The focal character of this vignette is Luhan Yang, former student of the Church lab and cofounder of eGenesis, a Boston-based startup using CRISPR to alter the pig genome in a way that will eliminate or minimize the risk of immune rejection. Pigs have a short gestation time, large litters, and human-sized organs, making them ideal for xenotransplantation.
Next, Davies introduces Church’s most highly publicized animal-editing endeavor, the “de-extinction” of woolly mammoths. While the resurrection of an extinct species is impossible because of irretrievable biological and social factors, researchers are using CRISPR to alter the genome of Asian elephants, giving these large animals the iconic dense hair and thick fat that would allow them to thrive in Siberia. The company Colossal, overseeing this brazen effort, exclaims that mammoths will “decelerate melting of the arctic permafrost” and “revert now-overshrubbed forests back into natural arctic grasslands.”
While the prospect of de-extinction remains arguably impractical, particularly for large mammals with long gestation times and complex fertilization processes, a third animal-editing application with ties to the Church lab has the potential for immediate and far-reaching consequences—gene drives. Gene drives involve the modification of DNA such that genetic changes are passed down to all future progeny. Davies chose to follow Kevin Esvelt, a pioneer in developing mosquito gene drives for the elimination of malaria and mouse gene drives for the control of Lyme disease. Esvelt is a quintessentially torn scientist, balancing the enormous global good of eradicating malaria with the infinite unknown of releasing millions of genetically modified insects.
A book on genome editing would be incomplete without xenotransplantation, de-extinction, and gene drives. These applications capture the imagination of readers, the concerns of policy makers, the interest of venture capitalists, and the worries of animal rights activists. But underneath this portion of the book is a prominent techno-optimism. In regard to gene drives, Davies states: “Could a gene drive spread across borders or to other species? Yes, perhaps. But isn’t biological warfare against one of the greatest killers of humankind worth the small risk?” Davies offers counter perspectives by referencing critics of gene drives from Burkina Faso in western Africa, but the sentiment that reverberates the loudest is the collective voice of scientists advocating animal editing to address some of society’s most pressing climate and health concerns.
To thoroughly examine the questions “is xenotransplantation the best way to address organ donor shortages” or “is de-extinction an effective or responsible way to improve environmental preservation or restoration?” would be outside the scope of this book. Regardless, by focusing on George Church and scientists a few degrees removed, the book’s tone takes on a spirit of “If not us, who? If not now, when?”
In many ways, editing a tomato is more difficult than a human. Tomatoes have 7,000 more genes, giving them a unique upper hand in genomic complexity. But the real hurdles exist beyond the biologic realm and into societal and political arenas. Public skepticism of genome-edited food paired with distrust of corporate motives has created uncertainty over when and how genome editing will eventually reach the dinner table, but scientists are confident that there is no question of if: ‘“The most profound thing we’ll see in terms of CRISPR’s effects on people’s everyday lives will be in the agricultural sector,’ predicts Doudna.” The main reason behind this confidence is the vital role that genome editing will play in adapting agriculture to a changing climate and the need to feed a growing population. Davies takes the reader through timely case studies of CRISPR’s potential role in safeguarding a threatened global food chain. But for Davies, and the reader, the arguably more interesting and critical questions pertaining to agricultural editing are the role of consumers and regulators.
Davies offers clear and compelling scenarios illustrating imminent, or even present, problems of drought and disease stressing already stretched food systems. The bacterial disease huanglongbing, is decimating orange groves in China, South America, and Florida. Panama disease is threatening to wipeout the iconic Cavendish banana. Relatively simple genetic modifications could give these staple crops another layer of protection against pathogens, but Davies points out that any change to their genomes could save them from disease at the expense of losing customer approval.
As for editing livestock, applications provide ethical and economic value, but confront burdensome regulatory hurdles. Davies explains the work of Alison Van Eenennaam, a proclaimed “livestock gene-editing evangelist,” who has genetically engineered dairy cows so they do not grow horns. This trait would allow farmers to do away with dehorning, the painful process of removing horns. To Van Eenennaam’s and other scientists’ frustration, these cows are regulated by the FDA, as “2,000-pound drugs,” rather than by the EPA like genetically engineered crops.
Similar to Davies’s earlier portrayal of genome editing applications across animal species, his tone regarding applications within agriculture is presented with a techno-optimistic hue. Davies is quick to note that he’s “not suggesting genome editing is the answer to feeding the planet, but it shouldn’t be stymied by overregulation or anti-GM hysteria.” He then exclaims that scientists “have failed miserably to persuade the public to embrace GM technology, leading an uphill road for CRISPR.”
As mentioned before, counter arguments raising the legitimate societal concerns of cost and access to seeds, lack of attention given to effective agroecological methods, and other factors impacting the embrace of GM technology are outside the scope of this book. Such topics warrant entire books themselves, but it’s important to note that the nuanced conversation around GM technologies is critical in achieving food justice, environmental sustainability, and economic fairness.
It’s fitting that the book’s final application of genomics research is centered around designer babies. The title of this chapter is “Volitional Evolution,” the idea that we can bend the arc of evolution in line with our will. One could argue that humanity has been performing this behavior for millennia. By simply selecting a sexual partner, a couple can steer evolution using one’s own volition. The resulting offspring from this primal act has characteristics from both parents, but the final outcome is still left to chance, one that many modern parents would like more control over.
Davies begins this section of the book with a frequently undiscussed reality of modern reproduction—the significant influence of private industry. Davies shares the story of GenePeeks, a now defunct startup that predicted which sperm donors would have an increased risk of creating an embryo with genetic disorders. There is a market for improving the chances of a healthy baby, which is why preimplantation genetic testing (PGT) is a growing business. The power of PGT is also a reason why genome editing is, in a vast majority of cases, unnecessary. By screening embryos, parents can prevent the inheritance of detrimental genetic mutations and would not need to use genome editing for corrective purposes.
Since PGT is an enticing market, companies like Genomic Prediction created their niche by offering parents the opportunity to choose between fertilized embryos based on height and cognitive ability, despite the scientific validity of such claims. Fertility clinics are sometimes considered the Wild West because of limited regulatory oversight. When mixed with capitalism, companies will continue to offer parents the opportunity to have children with increasingly “enhanced” traits, even if the legitimacy of such claims are dubious.
Davies ends this section with a point that is too often missed when discussing designer babies. “Genome editing won’t change society that much in the near future, but the prospect of genetic enhancement would only accentuate societal differences instead of trying to stem such inequalities.” Current methods of embryo editing require in vitro fertilization (IVF), a costly (~>$10,000) procedure and the “most common type” of assisted reproductive technology (ART). Given that in 2019, only 2.1 percent of all babies born in the United States were conceived using ART, it is unlikely that embryo editing will be widespread enough to have a species-level impact. Instead, this advanced reproductive technology will remain limited to wealthy individuals, further exacerbating health inequality on a generational level.
In comparison to Davies’s approach to describing animal and plant genetic engineering, his take on embryo editing feels more balanced, placing CRISPR within broader societal and technological considerations of reproduction. Unlike other authors who may dwell on the science fiction of designer babies, Davies ends on a pragmatic note, stressing the need to consider who will benefit from such applications.
The impact of CRISPR on genome editing mirrors that of the iPhone on communication. It is a disruptive technology, acting as a platform on which innovators can add new capabilities. And, like smartphones, the story of CRISPR is not finished. Editing Humanity captured CRISPR’s beginning, from the discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) by Francisco Mojica in the 1990’s up until a widely anticipated Noble Prize announcement in 2020. We may not know CRISPR’s full impact on society for decades, as the field is moving so rapidly that significant advancements have been made since the publication of the book.
A 2017 National Academies of Science report outlined “several criteria that should be met before allowing germline editing clinical trials to go forward.” While the use cases were limited to serious conditions, this report gave a “yellow light” to heritable genome editing under stringent oversight. JK cited this report as a rationale for moving forward, which critics argue was a blatant mischaracterization of the report. Regardless, JK’s actions demonstrated that better governance systems should be in place to monitor the global use of this rapidly evolving technology.
Over the last several years, dozens of national and international reports have sought to provide recommendations for the ethical and technical oversight of genome editing applications, many of which addressed germline editing. Since the publication of Editing Humanity, a WHO expert advisory committee on developing global standards for governance and oversight of human genome editing released two reports: a framework for governance and recommendations on human genome editing. As reported in September 2021, “[t]hese documents provided several case studies, covering the many different ways genome editing can be used to alter human cells,” including human embryos. Partially in response to the actions of JK, the reports “suggested ways to monitor unethical research on a global scale. For instance, the committee recommended creating a whistleblowing mechanism for scientists to report illegal activity.”
It remains to be seen how the WHO and country-level governments will operationalize these recommendations, but as Davies detailed, the onus is on countries to oversee the work of their researchers. JK was not the first researcher with germline genome editing aspirations and will not be the last.
Genome editing falls under the broader umbrella of gene therapy, the treatment of disease through the manipulation of DNA—a field that is rapidly growing. By the end 2022, over 2,000 gene therapies were either in preclinical research or later stage clinical testing, targeting dozens of different diseases, infections, and malignancies. Advancements in genome editing have spurred much of this growth and the first gene therapies using CRISPR technology are entering clinical trials and will likely be available to patients within the next few years.
In his book, Davies references several clinical applications of genome editing, including sickle cell disease, a genetic form of blindness, liver disease, and Duchenne muscular dystrophy. These disease targets were chosen as some of the first uses of CRISPR either for the relative simplicity of delivering CRISPR molecules to the cells of interest or the feasibility of creating a specific genetic change that would confidently slow, stop, or reverse the disease.
Since the publication of Editing Humanity, the FDA has approved human testing to evaluate the first use of CRISPR to treat HIV. Two CRISPR clinical trials for rare genetic diseases, transthyretin (ATTR) amyloidosis and hereditary angioedema, have demonstrated clinical benefits. Further along the clinical trial pipeline, CRISPR-based treatments for sickle cell disease and transfusion-dependent beta thalassemia are close to seeking regulatory approval from the U.S. FDA and the European Union’s European Medicines Agency.
While these clinical trials are fueling optimism, access remains an issue. As I noted recently,
Gene therapies are the most expensive drugs in the world. In 2022, a gene therapy for beta thalassaemia, a rare blood disorder, received regulatory approval in the US with a projected price tag of $2.8 million for a one-time infusion. This record was short lived as a month later, a gene therapy for a rare neurological disorder was priced at $3 million per treatment.
While prices remain high for now, technological innovation, corporate competition and new payment models are expected to lower costs and improve access to gene therapies in high-income countries. This is not the case for LMICs.
Regarding global access, I have observed the following:
The complex equipment, expert personnel and mature regulatory environments necessary to develop, test and administer gene therapies are largely insufficient or absent in low- and middle-income countries. This is partially why, in August 2022, there were approximately 1,000 open gene therapy clinical trials globally, yet fewer than five percent were recruiting in LMICs (not including China), with only four trials in Africa. Under this current trajectory, supply will never reach demand.
More than 300,000 babies are born each year with sickle cell disease, with nearly 75% of these births occurring in sub-Saharan Africa and almost 17% in the Arab-India region. As for HIV, in 2018, more than two-thirds of people living with HIV lived Africa and of that almost one-third live in South Africa. Addressing these enormous health burdens will require improved screening and better primary care, but emerging curative therapies should also be part of long-term strategic plans.
To close this global gap, the Bill and Melinda Gates Foundation is collaborating with Novartis, the U.S. National Institutes of Health (NIH) and other companies to develop single-shot gene therapy cures for HIV and sickle cell disease. Additionally, the Global Gene Therapy Initiative (GGTI), an alliance of clinicians, scientists, advocates and community members, aims to launch gene therapy clinical trials in Uganda and India over the next few years.
Editing Humanity covers a considerable amount of ground. Davies reaches back decades to the discovery of DNA’s double helix and looks far into the future, predicting the broad societal impact of genome editing. Yet, the final pages of the book may prove to be the most memorable. Davies quotes George Church saying “When people talk about the ethics of CRISPR, 90 percent of it should be, and probably is, about equal distribution of expensive technology.” It’s easy to be enthralled by what CRISPR can do, but in the end, it comes down to who or what are the gatekeepers to accessing this powerful technology.
