Last week, hundreds of international scientists convened at the Francis Crick Institute in London for the Third International Conference on Human Genome Editing. As they did at the first and second summits, which were held in Washington, DC, in 2015 and Hoin ng Kong, China, in 2018, leading researchers delivered their findings and discussed how they could be implemented.
The possibility of healing certain diseases by modifying their causative DNA sequences is becoming a reality. Treatment for sickle cell disease involving somatic genome editing is expected to receive regulatory approval in the United States later this year.
The supply of fair access to genome editing medicines, continued research to optimize delivery methods for genome editing devices, and the delivery of measures to facilitate conversations surrounding regulation, governance, and public and patient participation were all reoccurring themes.
As per The Conversation, in his opening remarks, the US Nobel laureate David Baltimore accurately highlighted that “new technologies continue to confront our society.” The introduction of CRISPR gene-editing technology, which is an abbreviation for “Clustered Regularly Interspaced Short Palindromic Repeats,” has reaffirmed this premise and sparked a global discourse on its associated ethical and regulatory challenges.
CRISPR technology has continued to develop since the last conference five years ago. It is impossible to document all of the scientific and ethical advancements made in CRISPR technology. They will be addressed in the context of the summit’s major themes: scientific advancements, accessibility, and the significance of public and patient engagement.
Several innovative strategies for genome editing were presented. David Liu, an American chemist, and scientist, published on the use of “prime editing” to treat genetic disorders such as Huntington’s disease and Friedreich’s ataxia. Prime editing generates a single-stranded DNA cut, as opposed to CRISPR’s double-stranded DNA cut. This renders it more adaptable and accurate for the deletion and insertion of certain genomic sequences.
The meeting learned about Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy. Later this year, the medication is anticipated to become the first CRISPR genome editing medicine to be approved.
There have also been reports of study utilizing CRISPR technology to cure diseases such as Duchenne muscular dystrophy, cancer, HIV/AIDS, heart and muscle disease, and innate immune disorders. As a therapy for myocardial infarction, the American molecular biologist Eric Olson reported success utilizing base editing to target CaMKII, a critical regulator of cardiac signaling, and restore heart function.
As research advances and therapies become available, questions regarding access to the technology arise. In addition to cost, access, and ownership, equity also includes research participation and production. This refers to the capacity for knowledge production, data sovereignty and collecting, access to the most up-to-date knowledge, cooperation opportunities, and infrastructure to support recruiting and clinical trials of new medicines.
Access concerns are especially pertinent to low- and middle-income nations, which may be hampered by systemic and structural imbalances. This inequality is perpetuated by policy and political landscapes, economic restrictions, and scientific racism.
Gautam Dongre, representing the National Alliance of Sickle Cell Associations India, emphasized the terrible access to treatment for persons with sickle cell disease in India: Our top aim is survival so that we can undergo gene therapy in the future. The summit also provided a forum for the experiences and concerns of individuals with genetic illnesses. These included insights about the function and value of public participation, such as patient advocacy groups, do-it-yourself community groups, and citizen juries.
Victoria Gray, the first recipient of Vertex Pharmaceutical’s CRISPR therapy for sickle cell disease, emphasized its life-altering effects in a remarkable presentation. Gray claims that her CRISPR-modified “super cells” have cured her, allowing her to live a life free of sickness. The enormous potential of CRISPR technology may be realized, but it must be made available to everyone.
How should the technology of CRISPR be regulated? This is a crucial inquiry. According to the summit’s organizers, somatic genome editing has made “amazing progress,” demonstrating its ability to “treat diseases that were formerly incurable.” More study is required to target additional diseases and improve our understanding of risks and unintended outcomes. “Somatic” genome editing (which produces non-heritable modifications) is distinct from germline and heritable genome editing (which makes heritable changes).
In gametes and embryos, for instance, fundamental research on germline genome editing, which is not intended for reproduction, is being conducted to investigate features of early development. However, the organizing committee determined that modifying the heritable human genome for reproductive purposes “remains inappropriate at this time.” In the absence of preclinical evidence of safety and efficacy, legal authorization, and stringent oversight and governance, this is the case.
The notion of “safe enough” was questioned; whose ethics should be used to establish this value judgment? Does the concept of safety extend beyond medically established dangers of bodily harm? It is notable that an individual’s risk tolerance and perception of safety are determined by their country, culture, socioeconomic standing, and life experiences.
The World Health Organization published a regulatory framework for human genome editing in 2021. This preserves its credibility as a model for the development of an acceptable regulatory structure. Although not being particularly prescriptive, it was designed to be applicable in any jurisdiction. Uganda intends to use the framework as a pilot project this year.
The organizing committee has asked for global effort to investigate equitable and cheap access pathways to genome editing treatments. Current global talks are far from conclusive, and they may never be, which reinforces the necessity for further dialogue at this summit. The pursuit of research, innovation, and collaboration continues.