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The Third International Summit on Human Genome Editing took place in London, UK on March 6-8, 2023. This Summit was significant in that they spent a significant time discussing sickle cell disease. You can access the agenda HERE (scroll down to the bottom).

One of the genome editing technologies, CRISPR, has the potential to cure diseases. Some people living with SCD have gone through CRISPR clinical trials and have been cured of the disease.

At the Summit, Victoria Gray was a special featured speaker because she was the first person living with sickle cell to be cured of the disease using this technology. She told a powerful story about her life before and after the gene therapy.

New SCDAA board member Dr. Melissa Creary was also among the featured speakers. She spoke from the patient perspective as someone contemplating the therapy as well as her concerns about equity and ethics related to the technology. Melissa briefly spoke with the media outlet Al Jazeera English about these things, and you can view that HERE.

You can see both Victoria and Melissa speak on Day One of the Summit HERE (starting at the time stamp 5:20:44).

MARAC member Dr. Julie Makani was on the organizing committee and spoke of gene therapy as one of the greatest advancements for sickle cell. The science, ethics, and patient perspectives were all discussed. Concerns about accessibility and hopes about the promise of a cure were covered throughout the day. Speakers on the ethics of gene therapy in sickle cell included Dr. Kofie Anie, psychologist and ethicist in one of London’s sickle cell centers, Daima Bukini representing the Muhimbili sickle cell center in Tanzania, and Dr. Alexis Thompson, who led sickle cell initiatives as former president of American Society of Hematology.

Here are some key take aways from the meeting that we’ve read about:

  1. Gene therapy progress is accelerating (“hitting the gas”), notably with sickle cell gene therapy going before the FDA – likely by the summer.
  2. Sickle cell disease is one of the pioneering success stories for gene therapy. Sickle cell suffering can be relieved by gene therapy, the data that we have collected so far proves it to us.
  3. The financial cost for gene therapy for people living with sickle cell is very expensive. Many are concerned that gene therapy access will be limited to the wealthy people in wealthy countries and that those who could help pay for the therapy for sickle cell, will not.  Some people are also concerned that the money spent for this therapy could be put to better use as standard treatment for people living with SCD.  Cost and access are long-term problems in sickle cell disease, and gene therapy accentuates these disparities.
  4. Although the technology is about the same, there is a deep ethical difference between gene therapy that affects just one person’s genes ( “somatic gene therapy”: not touching the genes of eggs or sperm) and gene therapy that changes genes that will be passed along to offspring (“heritable genomic editing”: changing the gene pool). The controversy whether to do gene therapy that changes to the gene pool includes removing gene mutations like the sickle gene, but also wealthy people creating “designer babies” (selecting to have family traits like hair color or athletic height).

If you’d like to read more about the Summit, you can click HERE.

NPR has exclusive rights to speak to Victoria Gray and has been covering the progress of gene therapy.  See two related articles that you might find interesting here:

Credits to Lewis Hsu and Melissa Creary