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news.The US Food and Drug Administration (FDA) has granted orphan drug designation for Korro Bio’s KRRO-110 to treat alpha-1 antitrypsin deficiency (AATD).
The therapy is currently undergoing a Phase I/IIa clinical study. Credit: National Cancer Institute on Unsplash.
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This investigational medicine is stated to be the “first” ribonucleic acid (RNA) editing development candidate from the company’s Oligonucleotide Promoted Editing of RNA (OPERA) platform.
Korro Bio chief medical officer Kemi Olugemo said: “Receiving orphan drug designation from the FDA underscores the growing need for new treatments for patients living with AATD.
“KRRO-110 has the potential to treat both liver and lung manifestations of AATD, and we are committed to addressing the unmet need and advancing this potentially best-in-class therapy for people living with AATD.”
It is currently being investigated in a Phase I/IIa REWRITE clinical study.
This two-part, single and multiple dose-escalating trial is aimed at assessing the therapy’s tolerability and safety in around 64 subjects which include the healthy adult population and “clinically stable” AATD individuals having the PiZZ genotype.
The first two single ascending dose cohorts’ dosing in healthy adult volunteers has been concluded. An interim readout of the study is anticipated in the second half of this year.
AATD is stated to be a genetic condition typically caused by a mutation in the SERPINA1 gene, leading to severe health issues such as pulmonary emphysema and hepatic cirrhosis.
KRRO-110 is tailored to utilise an endogenous enzyme to edit the SERPINA1 RNA, potentially restoring the normal production of AAT protein.
By repairing the amino acid codon, KRRO-110 claims to facilitate the clearance of protein aggregates from liver cells, offering a “clinically differentiated” benefit for the functioning of the liver. Additionally, it seeks to “preserve” its functioning by ensuring an adequate normal AAT protein amount is available.
