Poised to launch its first mar­ket­ing ef­fort, Al­ny­lam makes a big leap in­to CNS dis­eases and Alzheimer's

With its lead RNAi drug poised for an his­toric ap­proval, Al­ny­lam $AL­NY is now lay­ing plans to cre­ate a pipeline of CNS drugs that will tar­get some of the tough­est dis­eases that face re­searchers to­day — in­clud­ing Alzheimer’s, the Night King of dis­eases.

Al­ny­lam re­searchers are fol­low­ing up on a ro­dent study where they say they were able to de­liv­er a small in­ter­fer­ing RNA drug — or siR­NA —in­to the brain and spinal cord area. Us­ing in­trathe­cal in­jec­tion — cir­cum­vent­ing the blood-brain bar­ri­er — the biotech says that they were able to durably si­lence a dis­ease tar­get gene tran­script, of­fer­ing proof of prin­ci­ple ev­i­dence that they could suc­ceed where a long queue of con­tenders have failed.

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