“It is quite remarkable that this first [intravenous] CRISPR-based gene-editing effort has been so successful.”
that the treatment has still led to no major safety problems. And the blood levels of TTR—on average just 7% to 59% of their pretreatment levels, depending on which of four CRISPR doses people received—remained stable in a total of 15 people treated between 2 and 12 months ago.
The researchers don’t yet know whether the patients, who already have nerve pain and numbness throughout their body, will improve on neurological tests or whether the treatment may just stabilize the disease. Because peripheral nerves involved in pain and sensation take time to recover from the buildup of TTR, those tests are usually done 1 year after treatment, says Intellia Chief Medical Officer David Lebwohl.
The companies continue to enroll TTR amyloidosis patients with widespread nerve pain or numbness as well as people with a cardiac form of the disease. Intellia is also using a similar CRISPR treatment to edit a liver gene in people with hereditary angioedema, which causes severe swelling of limbs and airways.
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