Only Patient in N-of-1 CRISPR Trial for Duchenne Muscular Dystrophy Dies

A patient taking part in an FDA-sanctioned clinical trial assessing a CRISPR-based therapy for a rare form of Duchenne muscular dystrophy (DMD) has died.

Terry Horgan, the only patient in the CRD-TMH-001 trial of a novel CRISPR therapeutic, died last week. Horgan is the brother of the founder of Cure Rare Disease (CRD), a non-profit biotech that was spearheading the trial.