US EditionExperimental Gene Therapy Found to Slow Huntington's Disease Progression, Company Says
A clinical trial with 29 patients showed gene therapy slowed Huntington's disease progression by 75%, potentially extending life quality and expectancy, researchers said.
- UniQure announced this week that its experimental gene therapy AMT-130 slowed Huntington's disease progression by 75% over 36 months in a clinical trial at University College London.
- This trial follows earlier failures of antisense therapy targeting Huntington's disease and uses a one-time gene therapy to silence the faulty huntingtin gene causing the disorder.
- The trial treated 29 early-stage patients, with 17 receiving a high dose through a complex brain surgery lasting up to 18 hours, injecting a harmless virus to deliver microRNA that blocks the toxic huntingtin protein.
- Dr. Walid Abi-Saab stated on Wednesday that AMT-130 could dramatically change how Huntington's disease is treated, while other specialists described the findings as "spectacular" and "very promising."
- UniQure plans to seek FDA approval by 2026 despite treatment complexity and cost, and researchers are planning larger and preventative trials, signaling potential hope for altering Huntington's disease and similar disorders.
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A gene therapy success in Huntington's and Trump's autism announcement
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Huntington’s disease treated for first time using gene therapy
Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed. Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.
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