Alnylam Buoyed By Early Alzheimer’s Gene Silencing Success

Targets Early Disease-Causing Protein

Alnylam has unveiled Phase I data showing its RNAi candidate can eliminate the amyloid precursor protein in the brain – opening the door to treating Alzheimer’s and many more CNS diseases.

Alnylam building
• Source: Alamy

Alnylam has highlighted promising initial results from an investigational gene silencing therapy which suggest it can largely eliminate a key protein involved in Alzheimer’s disease, the first time such an approach has ever worked in the brain.

The company’s small interfering ribonucleic acid (RNAi) molecule named ALN-APP was shown to eliminate or ‘knock down’ a gene which...

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