Intellia CRISPR Therapy Cuts HAE Attacks by 87% in Phase 3
Intellia's lonvo-z reduced hereditary angioedema attacks by 87% in Phase 3, with 60% of patients attack-free and no serious safety signals reported.
rare disease
4 articles tagged "rare disease"
Sarepta Therapeutics Shares Surge on Rare Disease Drug Data
Sarepta Therapeutics shares rose 20% after releasing early clinical data for two rare muscle-wasting disease candidates, SRP-1001 and SRP-1003.
FDA Approves Kresladi Gene Therapy for Rare LAD-1 Disease
The FDA has approved Rocket Pharma's Kresladi for LAD-1, a rare immunodeficiency affecting roughly 25 US patients annually, marking a gene therapy milestone.
By Robert Kim
BioNTech Founders Depart, Senator Probes FDA Rare Disease
BioNTech co-founders plan to leave by 2026 to launch a new mRNA venture, while a U.S. senator investigates FDA's rare disease drug approval denials.
By James Liu