News of Alnylam's experimental RNAi drug thrilled both patients and scientists, who have been working for over a decade to create a drug which mutes misbehaving genes, having the potential to stop the production of proteins. 

RNAi-based therapeutics works by interrupting the production of disease-causing proteins, targeting the source of the disease. 

Many new and uncharted hurdles exist in order to turn RNAi into effective medicine, the most significant challenge according to the head of R&D at Alnylam is delivery of the drug to a relevant target organ.