The cost of gene therapy for once incurable diseases is too high, scientists warn

Medicine is on the brink of an era of highly precise genetic treatments, but urgent action is needed to bring down “extremely high” costs, experts told a global conference on human genome editing.

Clinical trials using new technologies such as Crispr show remarkable promise for treating many previously incurable diseases, researchers said at the International Summit on Human Genome Editing. The summit was convened by the Royal Society and Academy of Medicine of the United Kingdom, the National Academy of Sciences of the United States and the World Academy of Sciences.

But the conference’s organizing committee said at the end of the three-day session on Wednesday: “A global commitment to affordable and equitable access to these treatments is urgently needed.”

Unlike the last genome editing summit in 2018, which was haunted by the controversial revelation that He Jiankui had made hereditary mutations in the DNA of three embryos in China, the London event focused more on “somatic gene therapy” that benefits the individual. patients, but they are not passed on to the next generations.

“There has been amazing progress in somatic gene therapy using genome editing – in the science, the number of clinical trials started and the enthusiasm experienced,” said Summit Chair Robin Lovell-Badge.

Timothy Hunt, chief executive of the Alliance for Regenerative Medicine, which represents cell and gene therapy developers, said: “There are around 130 clinical trials underway worldwide to test gene editing technology for the permanent treatment and potential cure of rare and common diseases – and an impressive eight. trials in phase 3.”

Regulatory authorities in the US, EU and UK are expected to approve the first therapy based on Crispr, the most widely used genome-editing technology, later this year. The product, developed by the American Vertex Pharmaceuticals and Crispr Therapeutics, corrects sickle cell disease, i.e. a gene defect that causes a blood disorder.

But these treatments raise their own ethical problems, it was said at the conference. “The issue is one of fairness and justice,” Lovell-Badge said. “The prices are so high that it will be difficult to reach parts of the world where many people suffer from genetic diseases.”

No price has yet been announced for any of the upcoming genome-editing therapies, but analysts expect them to cost at least $2 million per patient.

The conference also discussed “human germline genome editing,” genetic changes in early embryos that affect all cells, including sperm and eggs, as described in He’s ill-judged and early experiment five years ago.

No one has attempted anything like it since. Scientists at the London conference said: “Heritable human genome editing remains unacceptable” because of the risks and ethical concerns of introducing mutations that are passed on to future generations.

But they said basic research using genome editing in human embryos should continue because it provides valuable scientific insight as well as information about how the technology can be safely used to correct genetic defects in the future.