Cost of gene treatments for once incurable diseases too high, scientists warn

Medicine stands on the threshold of an era of ultra-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 novel technologies such as Crispr are showing remarkable promise in treating a range of previously incurable diseases, researchers told the International Summit on Human Genome Editing. The summit was convened by the UK Royal Society and Academy of Medical Sciences, US National Academy of Sciences and World Academy of Sciences.

But the conference organising committee said at the end of the three-day meeting on Wednesday: “A global commitment to affordable, equitable access to these treatments is urgently needed.”

In contrast with the last genome editing summit in 2018, which was haunted by the controversial revelation that He Jiankui had made heritable mutations in the DNA of three embryos in China, the London event devoted more attention to “somatic gene therapy”, which benefits individual patients but will not be passed on to future generations.

“There has been amazing progress with somatic gene therapy using genome editing — in the science, in the number of clinical trials starting and in the enthusiasm we have seen,” said Robin Lovell-Badge, summit chair.

Timothy Hunt, chief executive of the Alliance for Regenerative Medicine, representing cell and gene therapy developers, said: “There are roughly 130 clinical trials globally to test gene editing technology to durably treat and potentially cure both rare and prevalent diseases — and an impressive eight trials in phase 3.”

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

But these treatments raise their own ethical issues, the conference heard. “The problem is that of equity and justice,” Lovell-Badge said. “The prices are so high that it is going to be difficult to reach parts of the world where many people are suffering from genetic diseases.”

No prices have yet been announced for any of the forthcoming therapies based on genome editing but analysts expect them to cost $2mn or more per patient.

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

No one is known to have attempted anything similar since then. Scientists at the London conference said: “Heritable human genome editing remains unacceptable” because of the risks as well as ethical doubts about introducing mutations that would be passed on to future generations.

But they said basic research using genome editing of human embryos should continue because it was giving valuable scientific insights as well as providing information about how the technology might be used safely to correct genetic errors in the future.

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