Last Week in Biomedicine | June 14–20 | Part 1 of 2: CRISPR HAE Cure, Narcolepsy Orexin Breakthrough

This week opened with one of the most significant gene-editing milestones in clinical medicine: a single CRISPR infusion cut hereditary angioedema attacks by 87 percent in a Phase 3 trial, published the same day in the New England Journal of Medicine. Plus a first-in-class drug that may reshape how we treat narcolepsy, and the first new oral antifungal class in over 20 years.

In Part 1 we cover:

1. Intellia Therapeutics — Lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (Phase 3)

2. Takeda Pharmaceutical — Oveporexton (TAK-861) for narcolepsy type 1 (Phase 3)

3. F2G / Shionogi — Olorofim for invasive aspergillosis (Phase 3)

The Intellia readout is the headline of the week. Hereditary angioedema causes sudden, painful and sometimes life-threatening swelling attacks, and current treatments require regular injections to suppress them. Lonvo-z is given once — a tiny package of CRISPR machinery shuts down the gene responsible for the attacks inside liver cells permanently — and 81 percent of patients were completely attack-free at one year. If this holds up, an entire category of chronic disease management could be replaced by single-dose curative gene

editing.

Part 2 drops Thursday morning. Subscribe at TrialBreaks.com.

#biomedicine #clinicaltrials #science #CRISPR #genetherapy #raredisease

#neurology #narcolepsy #orexin #infectiousdisease #antifungal #immunology #HAE

#aspergillosis #drugdevelopment