AUCKLAND DISTRICT HEALTH BOARD

CRISPR Therapy NTLA-2002 Shows Promise in Hereditary Angioedema Treatment

6 months ago

• NTLA-2002, a CRISPR-Cas9-based gene editing therapy, demonstrates potential as a one-time treatment for hereditary angioedema (HAE), reducing the need for daily medication. • Phase 2 study results published in the New England Journal of Medicine showed significant reductions in angioedema attacks and kallikrein levels in patients treated with NTLA-2002. • The study reported a mean percent change in the monthly attack rate from baseline through week 16 of -78% and -79.5% for 25mg and 50mg doses respectively. • Common adverse events associated with NTLA-2002 included headache, fatigue, and nasopharyngitis, with researchers noting limitations such as small sample size.