Treating genetic disease at scale with tRNA, with Michelle Werner

Описание: Genetic diseases are notoriously challenging to treat, especially when each condition requires a tailored approach. With over 10,000 known genetic disorders, developing individual therapies for each one has been an immense hurdle, particularly for rare diseases affecting small patient populations. But what if there was a way to address multiple conditions simultaneously, using a single, universal approach?



In today’s episode of the pharmaphorum podcast, Michelle Werner, CEO of Alltrna, discusses her company’s approach of leveraging transfer RNA (tRNA) to shift the paradigm in genetic medicine. This approach has the potential to offer hope to millions of patients with rare and ultra-rare diseases, bypassing traditional one-disease-at-a-time models.



Werner discusses how engineered tRNA works, how this technology slots into existing regulatory frameworks, and why this could be a game changer for pharmaceutical development.



You can listen to episode 249 of the pharmaphorum podcast (https://pharmaphorum.com/podcasts)  in the player below, download the episode to your computer, or find it - and subscribe to the rest of the series – on Apple Podcasts (https://podcasts.apple.com/gb/podcast...) , Spotify (https://open.spotify.com/show/1wbZ50I...) , Overcast, Pocket Casts, Podbean (https://pharmaphorum.podbean.com/) , and pretty much wherever else you download your other podcasts from.