Revolutionizing RNA therapeutics - Avidity Biosciences

We could not just change the lives of people living with myotonic dystrophy, but we could actually change the whole RNA field. That's pretty ambitious. Welcome to the RTW podcast. I'm your guest host, Piratip Ratoumswan, managing director and research analyst on the RTW asset selection team. Today, I have the privilege of speaking to Sarah Boyce, president and CEO of Avidity Biosciences, a company that is revolutionizing delivery of RNA therapeutics for patients living with debilitating rare genetic conditions. Over five years, Sarah transformed Avidity into a publicly traded leading genetic medicines company with three neuromuscular programs in late stage clinical development. Before that, Sarah was the president of Akcea Therapeutics, where she led the commercialization of oligonucleotide therapies. Sarah, it's great to have you here in New York at RTW's headquarters. Thank you very much for crossing the country to join us today. Thank you, and it's a pleasure to be here. Our audience know a lot about what you do and what Avidity does. But can you tell us a little bit more about Avidity's value proposition? Yeah. Avedi, we're trying to do two things. One is to revolutionize the RNA space, and two is to make a profound impact in people's lives. We have cracked and solved the challenge of delivery of cells and tissue types outside of the liver. Our first franchise is skeletal muscle where we now have three programs in the clinic. Two of those programs are for large rare diseases, population sizes of thirty to forty thousand people living with those diseases in The US. We did it for myotronic dystrophy. We've done the same thing for FSHD, where you really show and you understand biology, you can engage a target with an siRNA, and all the dots connect together from delivery to being able to engage with your genetic target to then being able to see functional changes. We have a franchise in precision cardiology, and, essentially, we are leading the field. We think that you are an optimistic person, and you're incredibly savvy. So what convinced you to join the team and lead Avedi? There was part of me going, what? They can deliver RNA to muscle cells. No one could do that. So I was intrigued. At that point, Avedi was private. We were 20 something people, preclinical. I met with Troy Wilson, and he was like, well, spend time with Art and see the data. Art and I have this very clandestine meeting because San Diego is a very small community. We were inside this golf club to make sure no one saw us, and he took me through the data. And I was like, so you're using an antibody. Like, why has no one thought of that? We all know antibodies are great at targeting certain cells. I've worked at platform technology companies previously and know that one of the great things is when you understand your safety really well, you know how to translate that from …