{
  "abstract": "Nexiguran ziclumeran is the first in-human CRISPR therapy designed to silence transthyretin production permanently in hereditary ATTR polyneuropathy. Remarkably, in a recent phase 1, open-label study it did exactly that: a single infusion knocked down serum TTR concentrations by around 90%, and this reduction persisted for 2 years. Clinical outcomes were more modest, however, with neuropathy scores remaining static for most patients. Safety outcomes were notable for infusion reactions and headache, and there were serious adverse events in almost one-third of patients (including a death from cardiac amyloidosis). And while the therapy progresses into Phase 3, A Fo Ben wonders what the future holds for these irreversibly genome edited human hepatocytes beyond the 2 year follow-up time. Nevertheless, for a disease long defined by rapid and progressive decline and either daily oral medication or repeated injections, the idea of a single session intervention is undeniably exciting.",
  "authors": [
    {
      "affiliations": [],
      "name": "A Fo Ben"
    }
  ],
  "title": "Carphology",
  "uid": "ebeff004-8f6f-5fed-a6b4-f06f2d9cfbb0"
}