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.