EP 94: Alnylam’s Dr Paul Nioi on how genetics is used in drug target discovery

“We’re pioneering the ability to detect genetic disease earlier, and actually intervene in a way that is going to help those patients.” In this jam-packed 45-minute episode, we’re joined by Dr Paul Nioi, Vice President of Discovery and Translational Research at Alnylam Pharmaceuticals and Chair of the Founders Board for Our Future Health. Join Patrick and Paul as they expose the two major reasons that 90% of clinical drug development fails, and why many believe that embedding genetics into target discovery and validation can increase the success rate of trials by 2-5 times. They also deep-dive into: the evolution of population genomics: from Iceland's DECODE to the UK Biobank examples of the value of recontact-by-genotype studies in supporting safety studies for novel targets, evidenced by the recontact of an ultra-rare homozygous loss-of-function carrier of the gene HAO1 the next chapter in population genomics that’s pioneering the ability to detect genetic diseases earlier the challenges of recontacting participants in biobanks and avoiding overwhelming the healthcare system, whilst responsibly handling patients’ sensitive data Alnylam’s work to develop therapeutics for genetic diseases based on RNAi, the discovery that was awarded the Nobel Prize in Medicine or Physiology in 2006 Tune in to benefit from Paul’s invaluable insights and expertise, which are coloured with practical examples from his career throughout. After listening to this 45 minute episode, you will: recognise the role of genetics in supporting the safety and efficacy of novel targets in clinical development understand the evolution of population genomics and what’s next understand how RNAi works, and how it differs from gene editing and other forms of next-generation therapies