Clinical-stage biopharmaceutical company Korro Bio Inc (Nasdaq:KRRO) announced on Friday that the US Food and Drug Administration (FDA) has granted orphan drug designation to KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency.

KRRO-110, an investigational RNA editing therapy, is designed to address both liver and lung manifestations of the disease.

Orphan drug designation provides regulatory incentives, including tax credits for clinical testing, user fee exemptions, and potential market exclusivity upon approval.

Korro Bio's chief medical officer, Kemi Olugemo, emphasised the urgent need for new treatment options for patients with Alpha-1 Antitrypsin Deficiency.

KRRO-110 is the first candidate from Korro Bio's OPERA platform and is currently being evaluated in the Phase 1/2a REWRITE study. The first two single ascending dose cohorts in healthy adult volunteers have been completed, with an interim analysis expected in the second half of 2025.

The FDA grants orphan drug designation to therapies targeting rare diseases affecting fewer than 200,000 people in the United States.

Korro Bio aims to advance KRRO-110 as a potentially best-in-class therapy for this underserved patient population.