Biopharmaceutical partner
Appears in 3 stories
Co-development and royalty partner on lonvo-z
On April 27, 2026, Intellia Therapeutics reported that its Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) — a one-time, in-body CRISPR gene-editing therapy for hereditary angioedema (HAE) — met its primary endpoint and all key secondary endpoints. In the 80-patient, randomized, double-blind trial, a single intravenous infusion of lonvo-z cut the rate of debilitating swelling attacks by 87% compared with placebo in the primary observation window (weeks 5 through 28). Sixty-two percent of patients who received the therapy were completely attack-free and had stopped all preventive medications in that period. The safety profile was clean: all reported side effects were mild or moderate, with no serious adverse events, and infusion-related reactions, headache, and fatigue were the most common complaints.
Updated 14 hours ago
Co-developer and co-marketer of Dupixent; 74% of revenue dependent on key biologic drugs
When the Food and Drug Administration (FDA) approved Dupixent for severe eczema in 2017, it was one drug for one disease. Nine years later, the same molecule has been approved for nine separate conditions, from asthma to a chronic fungal sinus infection that previously had no approved treatment at all. The latest approval, granted on February 24, 2026, covers allergic fungal rhinosinusitis (AFRS), a condition that traps patients in a cycle of repeated sinus surgeries. In a pivotal trial, Dupixent reduced the need for additional surgeries and steroid courses by 92%.
Updated Feb 24
Leading developer of DB-OTO gene therapy, seeking FDA approval
Children born profoundly deaf can now hear their parents' voices. A single injection of gene therapy into the inner ear has restored hearing in dozens of patients with genetic deafness, moving some forms of lifelong hearing loss from permanent disability into the treatable category. The effect is rapid—most patients recover hearing within weeks—and sustained over at least two years of follow-up.
Updated Jan 9
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