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
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
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