Alnylam's experimental Huntington's therapy reduces mutant protein in early trial

Alnylam Pharmaceuticals reported early data from a small study of its gene‑silencing drug for Huntington's disease.

The investigational agent, brezosiran, was administered at several dose levels. Patients receiving the highest dose showed a reduction of mutant huntingtin protein in cerebrospinal fluid of roughly 60% to 64%, depending on the assay used.

The company described the safety profile as encouraging and said it will launch a placebo‑controlled trial next year to determine whether the protein knockdown can slow disease progression.

These findings add to growing interest in RNA‑interference approaches for neurodegenerative disorders, though it remains to be proven whether the biomarker changes will translate into clinical benefit.