TL;DR
Hereditary transthyretin amyloidosis is an inherited disease in which a misfolded protein made in the liver slowly damages nerves, and in another form of the disease, the heart. Intellia and Regeneron are testing a different kind of answer: nex-z, a CRISPR gene-editing drug given once, as a single infusion, instead of a treatment taken for life. Their make-or-break Phase 3 trial in the nerve form of the disease has now finished enrolling about 60 adults, half on the drug and half on placebo, which starts an 18-month clock to the main result. The news comes from the company's announcement as relayed by a financial news site, a secondary report. If the readout lands, a single visit could one day stand in for years of ongoing treatment for families who carry this gene. It is also a test of a bigger idea: if one infusion can switch off a faulty gene for good, the same approach could work for many other inherited diseases that are now treated for life. To see our odds on when a one-time CRISPR drug for this disease reaches patients, and the timeline of breakthroughs that leads there, subscribe.
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