Alnylam drug found effective in treating rare genetic disease

Alnylam Pharmaceuticals Inc said an early-stage trial of its gene-based therapy showed the drug was effective in suppressing a protein that causes a rare organ-damaging hereditary disorder.

Alnylam shares rose 14 percent to $42.80 in premarket trade.

The study was testing a subcutaneously administered version of the drug, ALN-TTR, to treat genetic disorder amyloidosis, which is caused by deposits of protein in tissues and organs.

The therapy is based on Alnylam’s gene silencing technology that works by “switching off” certain disease-causing genes and is widely believed to hold immense potential for treating diseases ranging from cancer to HIV.

(Reporting by Esha Dey in Bangalore; Editing by Saumyadeb Chakrabarty)

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Reuters