Korro Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency

 Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).

KRRO-110 is the first RNA editing development candidate from Korro’s proprietary OPERATM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the first two single ascending dose cohorts in healthy adult volunteers has been completed, and an interim readout is expected in the second half of 2025.

https://www.globenewswire.com/news-release/2025/03/14/3042908/0/en/KRRO-110-Receives-Orphan-Drug-Designation-from-U-S-FDA-for-Alpha-1-Antitrypsin-Deficiency.html