Rapid Analytics And Next Generation Sequencing

By Mike Paglia, Chief Technology Officer of BaseCamp

Advancements in gene editing technologies rapidly transform the landscape of genetic medicine, with RNA optimization emerging as a critical factor in enhancing therapeutic outcomes. Explore in vivo strategies for refining RNA design and delivery to maximize gene editing efficiency specifically in liver tissue—a key target for treating metabolic and genetic disorders.

Leveraging ElevateBio’s integrated technology stack, including RNA-guided nucleases, base editors, and advanced delivery systems like lipid nanoparticles (LNPs), researchers are pushing the boundaries of precision and efficacy. The article delves into how next-generation sequencing (NGS) analytics are being used to validate editing outcomes, ensure safety, and streamline development workflows. It also highlights the importance of sterility and identity testing, showcasing how custom target capture technologies and automated pipelines are accelerating the path from concept to clinic.

By combining robust analytical development with scalable cGMP manufacturing, these RNA optimization strategies not only improve editing accuracy but also reduce production timelines and costs. This preview sets the stage for a deeper look into how RNA engineering is unlocking new possibilities in liver-targeted gene therapies and redefining the future of personalized medicine.