No drug can slow Huntington's yet — a gene-silencing shot just cut its toxic protein by about two-thirds

Huntington's is an inherited brain condition: a faulty gene tells the body to make a harmful form of a protein called huntingtin, and that protein slowly damages the brain. There is still no approved medicine that slows the disease. In an early trial reported today, a single dose of an experimental treatment called brezosiran — given into the spinal fluid and designed to switch off the instructions for making that protein — cut the harmful protein by 60 to 64% on average at the highest dose tested, and by up to 83% in one person. The 48-person study, run in Canada, Germany and the UK and presented in Krakow, Poland, found the treatment was tolerated encouragingly well over up to 12 months, with a possible twice-a-year dosing pattern. A larger placebo-controlled trial is planned for 2027 to test whether lowering the protein actually slows the disease — the question that matters most.

SDG 3 · Good health and well-being — Mechanism: tech innovation