Intellia Therapeutics announced on Monday that a single dose of its CRISPR-based gene editing treatment, lonvo-z, dramatically reduced swelling attacks in patients with a rare genetic disorder during a Phase 3 clinical trial. The results position lonvo-z as a potential second CRISPR-based therapy approved for medical use.
The therapy, lonvo-z, would follow Vertex Pharmaceutical’s sickle cell treatment, Casgevy, as the second CRISPR-based medicine approved by regulators. Intellia has already begun a rolling submission with the U.S. Food and Drug Administration (FDA), which could expedite the approval process.
Lonvo-z represents the first in vivo CRISPR treatment, meaning it edits patients’ DNA directly inside the body, rather than in a lab-grown sample.
Phase 3 Trial Results: Lonvo-z Cuts Attack Rates by 87%
In the trial involving 80 patients with hereditary angioedema (HAE), those who received lonvo-z experienced an 87% reduction in swelling attack rates compared to the placebo group. Additionally, over 60% of patients treated with lonvo-z remained entirely free of attacks during the study period, compared to just 11% in the placebo group.
The trial’s findings highlight the potential of lonvo-z to transform treatment for HAE, a rare and debilitating condition characterized by unpredictable and painful swelling episodes.
Related articles
FDA Drug Center Acting Director Tracy Beth Høeg Departs Amid Leadership Changes
WASHINGTON — The acting director of the Food and Drug Administration’s drug center, Tracy Beth Høeg, is leaving the agency, according to three sources...
U.S. Pressures Germany on Drug Prices Amid FDA Restructuring Concerns
And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about we...
Mail-Order Abortion Pills Remain Legal Amid Legal Uncertainty
In a discussion about the best fruits, my STAT colleague Alexa Lee and I agreed that peaches have been mid in recent years. Is this just a New York Ci...
FDA Rejects Melanoma Drug RP1 Amid Leadership Controversy Under Makary
The FDA’s recent decision to withhold approval of a new skin cancer treatment fell like a hammer on doctors who treat melanoma and patients who saw th...
Dr. Makary's Exit and the Future of Cassidy's Senate Seat: Key Health Policy Shifts
This week’s episode of “The Readout LOUD” is all about health politics. We bring on FDA reporter Lizzy Lawrence to discuss Makary’s departure — why he...
Who Will Lead the FDA Next? The Search for a New Commissioner Heats Up
You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to...
BeOne Secures FDA Approval for Novel Lymphoma Cell Therapy, Advancing Autoimmune Treatment Pipeline
Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. Biotech’s cell therapy boo...
Biogen’s Tau-Targeting Alzheimer’s Drug Shows Promise in Phase 2 Trial; Takeda to Cut 4,500 Jobs by 2026
Rise and shine, everyone, another busy day is on the way. And it is getting off to a pleasant start here on the Pharmalot campus, where clear blue ski...