Intellia suggests ATTR amyloidosis gene editing treatment has over a year of durability as it gears up for PhIII
Intellia on Thursday presented new results for its gene editing treatment in 65 patients with ATTR amyloidosis — a condition in which misfolded proteins build up in the body — and the data suggest that the treatment can lower levels of the protein in the blood by around 90%.
In 62 patients who received a one-time dose of at least 0.3 mg/kg of NTLA-2001, the median reduction in levels of transthyretin, the protein that gets misfolded, was 91% after one month. And in 29 patients who were dosed at least one year ago, that reduction has held, according to Intellia.
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