Functional
Regenxbio Reports Consistent One-Year Benefit for DMD Gene Therapy RGX-202
Regenxbio; DMD; Duchenne muscular dystrophy; RGX-202; gene therapy; AFFINITY DUCHENNE trial; clinical trial; microdystrophin; biomarker; phase I/II; functional data
Intellia’s CRISPR Therapy NTLA-2002 Shows 81% Reduction in Hereditary Angioedema Attacks in Phase 2 Study
CRISPR therapy, Intellia Therapeutics, NTLA-2002, Hereditary Angioedema (HAE), Gene editing, Phase 2 study, Swelling attacks, Functional cure