Rocket’s Gene Therapy Trial Paused, Boltz-2 Open Source, Plasma Swap Rewinds Aging

A gene therapy clinical trial for Danon disease run by Rocket Pharmaceuticals has been suspended following the death of a patient. MIT and Recursion have released Boltz-2, an open-source tool that predicts molecular binding affinity at newfound speed and accuracy, aiming to democratize commercial drug discovery. Intellia Therapeutics saw its stock nosedive late last week after a patient in its Phase III trial of the CRISPR-based therapy experienced non-lethal liver toxicity. In other news, a therapeutic plasma exchange regimen combined with intravenous immunoglobulin reduced some biomarkers of biological age in humans. Sanofi has agreed to acquire Blueprint Medicines for up to $9.5 billion, and a universal CAR T cell therapy was produced by applying CRISPR gene editing to cells donated by healthy individuals.  Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news.   Listed below are links to the GEN stories referenced in this episode of Touching Base  Danon Disease Patient Dies in Rocket Gene Therapy Trial By Alex Philippidis, GEN, May 27, 2025  Boltz-2 Released to Democratize AI Molecular Modeling for Drug Discovery By Fay Lin, PhD, GENEdge, June 6, 2025  StockWatch: Intellia Stumbles on News of Patient’s Severe Liver Toxicity By Alex Philippidis, GEN, May 30, 2025  The State of CRISPR Summit Registration https://bit.ly/CRISPR25  Plasma Exchange in Humans Reduces Biological Age by Over Two Years  GEN, June 1, 2025   Sanofi to Acquire Blueprint Medicines for Up to $9.5B By Alex Philippidis, GEN Edge, June 2, 2025  "Universal" CAR-T Immunotherapy Targets Relapsed/Refractory Blood Cancers GEN, May 30, 2025  Hosted on Acast. See acast.com/privacy for more information.