Christopher Cole

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Christopher Cole is Associate Editorial Director for BioPharm International®, Pharmaceutical Technology®, and Pharmaceutical Technology Europe™.

Articles

Personalized CRISPR Therapy Successfully Treats Infant With Rare, Incurable CPS1 Deficiency

Christopher Cole

May 16th 2025

A pediatric patient with a rare genetic disorder safely received a personalized CRISPR therapy, marking the first known case of a personalized CRISPR-based medicine administered to a single patient.

Transfer RNA (abbreviated tRNA and formerly referred to as sRNA, for soluble RNA with RNA strand in the cell 3d rendering | Image Credit: © Love Employee - stock.adobe.com

Early Data Show Potential of Tevard’s tRNA Therapy in Duchenne Muscular Dystrophy

Christopher Cole

May 15th 2025

Tevard's tRNA therapy appears to restore full-length dystrophin and motor function in a Duchenne muscular dystrophy model, showing promise for nonsense mutation treatments.