Embracing the Power of RNAi Therapeutics: Akshay Vaishnaw, Alnylam Pharmaceuticals

·36 min·2 clips

Alnylam’s first major breakthrough was delivering siRNA to the liver in mice and monkeys.

1. The Real Chemistry Podcast episode centers on Akshay Vaishnaw and Alnylam Pharmaceuticals’ RNA interference therapeutics work. 2. Aaron Strout hosts the conversation, and Vaishnaw serves as Alnylam’s chief innovation officer, bringing experience from clinical medicine through research and company leadership. 3. The episode asks what RNAi is for, how Alnylam turned it into medicines, and why the technology still matters after years of setbacks. 4. Vaishnaw says he was born in India, emigrated to the UK at age eight, and became interested in science after watching Apollo missions and reading Scientific American and New Scientist. 5. He explains that medical school, a PhD, and training in internal medicine, rheumatology, and immunology led him into research and then a 1990s fellowship in the US. 6. He says Biogen in 1998 felt pivotal because molecular genetics and molecular biology were beginning to reshape human disease treatment. 7. Vaishnaw joined Alnylam at the end of 2005 after a seven-year period at Biogen and has since worked across clinical, medical, R&D, and innovation roles. 8. He describes Alnylam growing from roughly employee number 50 to more than 2,500 people. 9. He says that growth gave him successive remits under senior leaders, including the recently retired CEO John Maraganore. 10. He explains RNA interference as a natural process in which cells control RNA levels so proteins are made in the right amounts for changing conditions. 11. He says Alnylam’s scientific founders showed in 2001 that RNAi occurs in human cells, and he notes that Fire and Mello received the 2006 Nobel Prize for Physiology or Medicine. 12. He details the delivery challenge: small interfering RNA is about 14,000 Daltons, cannot be taken orally, breaks down in blood, and must still enter cells. 13. He says the 2005–2006 lipid nanoparticle breakthrough enabled siRNA delivery to the liver in mice and monkeys. 14. He describes the TTR program starting around 2007, with the first IND filed in 2010 and human TTR knockdown shown in 2012. 15. He says Onpattro was approved in 2018 for TTR neuropathy after about 16 years from company founding and 10 years from the concept. 16. He recounts setbacks including two rounds of layoffs, trading under cash, partner exits, and a 2010–11 result in which only one patient out of 27 showed TTR reduction. 17. He says John Maraganore asked him whether he believed in the science and the people, which helped him recommit during that bleak period. 18. He says Alnylam’s culture emphasizes commitment to people, and he points to work on gender balance, pay equity, and inclusion as part of that culture. 19. The interview stays conversational and detailed, with Strout pressing for plain-language explanations, personal stories, and forward-looking examples. 20. Listeners interested in genetic medicines, biotech leadership, and drug development will get the most from this episode, while people wanting a short celebrity-style interview may skip it.

Listen to the show on

Apple Podcasts Spotify

Play clips from this episode More from The Real Chemistry Podcast