Korro Bio, Inc. (KRRO), a clinical-stage biopharmaceutical company, announced Friday that it has received orphan drug designation from the U.S. Food and Drug Administration or FDA for its investigational medicine, KRRO-110, for the treatment of Alpha-1 Antitrypsin Deficiency or AATD.
This designation recognizes the potential of KRRO-110 to treat both liver and lung manifestations of AATD, a rare genetic disorder.
The drug is currently being evaluated in the Phase 1/2a REWRITE clinical study, with the first two single ascending dose cohorts in healthy adult volunteers having been completed.
An interim readout of the data is expected in the second half of 2025.
Orphan drug designation provides various development incentives, including tax credits for clinical testing and potential market exclusivity upon FDA approval.
KRRO-110 is the first RNA editing development candidate from Korro's OPERATM platform, with the potential to offer a differentiated treatment for AATD patients.
This milestone marks an important step for Korro as it advances its RNA editing platform in the development of genetic medicines for rare diseases like AATD.
Currently, KRRO is trading at $24.59, down by 0.28 percent on the Nasdaq.
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