The Drug Discovery World Podcast

DDW Highlights: 14 April 2026

In this episode, Bruno Quinney highlights three major developments: the discovery of a new recessive neurodevelopmental disorder linked to mutations in the non‑coding gene RNU4‑2, a first‑in‑human trial of personalized CAR‑T cell therapies for hard‑to‑treat solid tumors in children, and FDA approval of Eli Lilly’s oral GLP‑1 pill (Foundio) for obesity along with a new Alzheimer’s collaboration between Lilly and AC Immune. The RNU4‑2 work, using saturation genome editing, maps over 500 variants and clarifies how different mutations cause distinct diseases, offering faster diagnosis and therapeutic insights. The CAR‑T trial aims to improve survival and reduce long‑term side effects for pediatric solid‑tumor patients, while the GLP‑1 approval expands weight‑loss options and underscores ongoing innovation in metabolic disease treatment.