RNAi has changed the way that rare diseases are treated - is it about to do the same for more common conditions?

Alnylam was the very first company to translate RNAi from Nobel Prize winning science into a commercially scalable platform. Founded in 2002, the company today has five different RNAi therapies on the market and a pipeline that continues to expand. While their early focus was on rare disease, today they are expanding into more common conditions, partnering with top pharma companies to bring next-generation therapies to market.

This week I sat down with Paul Nioi, Senior Vice President of Research at Alnylam. Paul has more than 20 year’s experience in biotech and biopharma, and he walked me through Alnylam’s mission and place in the biotech ecosystem, the science of RNAi, the early success treating metabolic disease, and exactly where their platform is expanding into now and in the near future. Paul explains the capacity of RNAi to address neurological and infectious disease, the value of large-scale genomic initiatives, and how he sees drug discovery evolving in the years ahead.

01:29               Meet Paul Nioi

08:56               Alnylam, its mission, and its science

14:35               Two types of partnerships: top pharma, and genomic initiatives

22:34               RNAi therapies on the market for rare disease

27:29               Expanding into more common conditions

32:17               INHBE mutations and cardiometabolic disease

35:01               Working with Roche to target hypertension

38:58               Looking forward: Alnylam’s platform in the near future

41:49               What role for national genomic initiatives?

45:16               RNAi’s place in a future of precision medicine

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To dive deeper into the topic: 

• mRNA, RNAi, circRNA, ASOs: A comparative guide to RNA therapeutics
• Six biotech companies leading the charge in hemophilia treatment
• Episode 159: Day One DNA - How and why the UK is betting on whole genome sequencing for every newborn