World News | Somatic genome-editing therapies are becoming a reality – but debates continue over ethics, equitable access and governance

Adelaide, March 11 (Dialogue) Hundreds of experts from around the world gathered this week at the Francis Crick Institute in London for the third International Summit on Human Genome Editing.

As with the first and second summits, held in Washington DC in 2015 and Hong Kong in 2018, leading research experts shared their findings and discussed how they could be used.

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The prospect of curing some diseases by changing the part of DNA that causes them is becoming a reality. A somatic genome-editing treatment for sickle cell disease will receive regulatory approval in the United States later this year.

“Delivery” is a recurring issue: delivery of equitable access to genome-editing therapies, ongoing research to optimize delivery systems for genome-editing devices, and delivery measures to facilitate discussions on regulation, governance, public and patient engagement.

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American Nobel laureate David Baltimore aptly noted in his opening remarks that “new technologies continue to challenge our society”. The advent of CRISPR gene-editing technology (short for “Clustered Regularly Interspaced Short Palindromic Repeats”) has reaffirmed this claim, sparking a global conversation about its attendant ethical and regulatory issues.

Five years after the last summit, CRISPR technology continues to mature. Capturing all scientific and ethical developments in CRISPR technology is an insurmountable task. These issues will be addressed with reference to the key themes raised during the summit – scientific developments, accessibility and the importance of public and patient engagement.

scientific development

Many new advances in genome editing technology are presented.

American chemist and biologist David Liu reports the discovery of using “prime edits” to treat genetic diseases such as Huntington’s disease and Friedreich’s ataxia. Unlike CRISPR, which makes double-strand cuts in DNA, primer editing induces single-strand cuts. This makes it more versatile and precise in targeted deletions and insertions of genetic sequences.

The summit heard about Vertex Pharmaceutical’s CRISPR-based treatment for sickle cell disease. The therapy is expected to become the first CRISPR genome-editing therapy approved later this year.

There have also been reports of studies using CRISPR technology to treat diseases such as Duchenne muscular dystrophy, cancer, HIV/AIDS, heart and muscle disorders, and congenital immune deficiencies. American molecular biologist Eric Olson reports the successful use of base editing to target CaMKIId, a central regulator of cardiac signaling, to restore cardiac function as a treatment for myocardial infarction.

fair access

As research progresses and treatments emerge, questions arise about equitable access to technology.

Equity extends beyond considerations of cost, access and ownership to research participation and output. This refers to knowledge production capabilities, data sovereignty and collection capabilities, access to the latest knowledge, opportunities for collaboration and infrastructure to facilitate the recruitment and trials of new therapies.

Access issues are particularly relevant to low- and middle-income countries, which may be affected by systemic and structural inequalities. Policy and political landscapes, economic constraints, and scientific racism further exacerbate this inequality.

Gautam Dongre, who represents the National Federation of Sickle Cell Organizations in India, described the reality of sickle cell patients in India, who struggle to access treatment:

“Our priority is to be alive and receive gene therapy in the future.”

Patient Perspectives and Public Engagement

The summit also provides a platform for those who have lived with genetic disorders to express their experiences and concerns. This includes insights into the role and utility of public engagement, such as patient advocacy groups, do-it-yourself community groups, and citizen juries.

A memorable presentation by Victoria Gray, the first recipient of Vertex Pharmaceutical’s CRISPR therapy for sickle cell disease, highlighted its life-changing impact. Gray said her CRISPR-modified “supercell” cured her, allowing her to live a disease-free life. The enormous potential of CRISPR technology is within reach, but it is important that it is available to all.

conclusion

How should CRISPR technology be regulated? This is a key question.

As summit organizers noted, “remarkable progress” has been made in somatic genome editing, demonstrating its ability to “cure once incurable diseases.” Further research is needed to target more diseases and enhance our understanding of risks and unintended consequences.

“Somatic” genome editing (producing non-inheritable changes) differs from germline and heritable genome editing (producing heritable changes).

Basic research on germline genome editing is underway, for example, to explore aspects of early development in gametes and embryos other than for reproductive purposes. However, the organizing committee concluded that heritable editing of the human genome for reproductive purposes “remains unacceptable at this time.” This is because of a lack of preclinical evidence of safety and efficacy, legal mandates, and strict oversight and governance.

The notion of “safe enough” is called into question – whose morals should be applied to make such value judgments? Does the concept of safety extend beyond the medically defined risk of bodily harm?

It is worth noting that risk tolerance and sense of security depend on an individual’s status, culture, socioeconomic status and life experiences in their country.

In 2021, the World Health Organization publishes a framework for governing human genome editing. This retains its authority as an example of a pathway towards an appropriate regulatory framework. While not overly prescriptive, it is designed to accommodate implementation in any jurisdiction. This year, Uganda plans to implement the framework as a pilot project.

The organizing committee called for global action to explore measures for equitable and affordable access to genome-editing therapies. The ongoing global discussions are far from complete, and perhaps never will be, reinforcing the need for collective dialogue to advance this summit. And continue to research, innovate and collaborate. (dialogue)

(This is an unedited and auto-generated story from a Syndicated News feed, the content body may not have been modified or edited by LatestLY staff)