Serendipity and siRNA: Julia Alterman on Expanding RNA Therapeutics

Welcome to Dose of Discovery: a dose of breakthrough medical discoveries, delivered with your morning coffee. (Coffee not included.)In this first episode, hosts AJ and Søren speak to Julia Alterman, assistant professor at the RNA Therapeutics Institute at UMass Chan Medical School, about how siRNA technology has evolved from a useful laboratory research tool into a promising therapeutic modality.Their conversation explores innovations in siRNA chemistry and how these changes have enabled researchers to craft potent siRNA drugs. Julia talks about her unlikely beginnings as a theater major before finding her way into biology – eventually leading her into the biotech industry and academia. Along the way, she discusses the challenges of delivering siRNAs beyond the liver into harder-to-reach tissues such as the brain and muscle, and the serendipitous discovery that helped launch Atalanta Therapeutics.In this episode:04:23 – Explainer: What are siRNAs?08:42 – Meet Julia Alterman12:39 – Julia’s history in industry16:33 – UMass and a PhD pivot18:28 – siRNAs versus ASOs21:39 – siRNAs versus CRISPR28:33 – A serendipitous breakthrough38:02 – Baby steps and big leaps42:10 – Stability and the therapeutic window46:27 – The importance of strong science communication50:55 – Scientists and VC funding54:28 – Wrap up and reflectionsAbout the guestJulia Alterman is an assistant professor at the RNA Therapeutics Institute at UMass Chan Medical School, where she develops new siRNA therapies for genetically defined diseases. Her work spans RNA chemistry, delivery, and pharmacology. Her earlier research into siRNA scaffold was licensed to Atalanta Therapeutics for the treatment of neurodegenerative disease.https://www.umassmed.edu/khvorovalab/People/faculty/Julia-Alterman/Dose of Discovery is cohosted by Søren Hough and Ayokunmi Ajetunmobi (AJ) and produced by Suze Cooper.LINKS:RNA Fact Sheet from the National Human Genome Research InstituteBioXconomy’s coverage of the Alterman and Khvorova labs:"siRNA backbone modifications boost therapeutic potential, study finds" by Nnenna Ohaka (Exploration of exNA)"Cutting-edge siRNA therapy improves ALS symptoms, survival in mice" by Søren Hough (Exploration of divalent siRNAs)"Long-lasting siRNAs could help cancer patients and older people resist muscle loss" by Søren Hough (Including a photo of a Belgian blue cow)"Oligo-based therapies lead charge against prion disease" by Nnenna OhakaRead more at Bioxconomy, sign up to our newsletter, and don’t forget to check out our upcoming events. You can find a fully annotated transcript of the episode here. Hosted on Acast. See acast.com/privacy for more information.