Scaling CRISPR For Rare Disease With Aurora Therapeutics' Dr. Edward Kaye скачать в хорошем качестве

Scaling CRISPR For Rare Disease With Aurora Therapeutics' Dr. Edward Kaye

On episode 124 of Cell & Gene: The Podcast, Aurora Therapeutics' CEO Dr. Edward Kaye discusses the company’s strategy for translating CRISPR gene editing into scalable, commercially viable medicines for rare diseases. Aurora is initially targeting phenylketonuria (PKU) using a platform approach that leverages shared components, such as lipid nanoparticles and base editors, while customizing guide RNAs for specific mutations. Dr. Kaye explains how regulatory flexibility and optimized manufacturing could make it possible to treat many genetic variants efficiently and cost-effectively. Ultimately, Aurora aims to build a repeatable model that expands gene editing access to larger rare-disease populations while keeping patients at the center of development. Email Erin at erin.harris@cellandgene.com with topics you'd like us to cover in future episodes. -- #celltherapy #genetherapy Subscribe to the podcast! Apple - https://podcasts.apple.com/us/podcast... Spotify - https://open.spotify.com/show/78NSjRu... YouTube -    • Cell & Gene The Podcast