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The deaths of two children who received CRISPR therapies in China have rattled scientists who work on similar genetic medicines in the US, adding new fuel to a yearslong debate about the danger of the gene therapy field’s most-used delivery method.
At the heart of the safety concerns are engineered viruses called adeno-associated viruses, or AAVs, that were used to shuttle the gene editing treatments into the body. While they’re the workhorses of gene therapy, more than 20 people around the world have died after receiving AAV-based treatments, many of which were given at doses that some scientists now view as dangerously high.
The two children’s deaths, which occurred over a year ago, were only brought to light recently. And due to the limited information about the incidents in China, it’s difficult to fully incriminate AAV or to exonerate the CRISPR gene-editing tool.
But many of the 15 genetic medicine experts that Endpoints News spoke with said the few details available about the deaths fit the recurrent pattern of problems seen with AAVs. And the tragedies are reigniting bigger questions...