Groundbreaking therapy could slow Huntington’s disease progression

Researchers may have discovered a potential treatment option in patients with Huntington’s disease.

In a press release, the researchers reported positive results from their phase I/II trial, which examined the efficacy of the gene therapy AMT-130 — which works by diminishing the activity of the HTT gene responsible for the development of Huntington’s disease. During a magnetic resonance imaging-guided procedure, the therapy is administered via catheters inside the brain. The 29 patients involved in the trial were divided into two groups — a low- and high-dose treatment group — and subsequently compared with patients who received standard care.

After a follow-up of three years, the researchers discovered that the patients who received AMT-130 demonstrated a 75% lower rate of Huntington’s disease progression, declines in neuronal damage and greater preservation of daily function.

As a result of the findings, the researchers plan to submit an approval application to the U.S. Food and Drug Administration in 2026.

Read more: Live Science

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