KRRO-110 is developed from Korro’s ribonucleic acid editing platform. Credit: Gorodenkoff/Shutterstock.

Korro Bio has secured approvals in Australia to commence the two-part Phase I/IIa REWRITE trial of its investigational product, KRRO-110, aimed at treating alpha-1 antitrypsin deficiency (AATD).

The approvals were granted by the Australian Bellberry Human Research Ethics Committee (HREC) and the Therapeutic Goods Administration (TGA).

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The single and multiple-dose escalation trial will involve 64 participants, including both healthy adults and AATD patients with the protease inhibitor ZZ (PiZZ) genotype. It is set to assess KRRO-110’s tolerability, pharmacodynamics, pharmacokinetics and safety.

Secondary and exploratory endpoints of the REWRITE study are set to focus on pharmacokinetic and pharmacodynamic parameters which may assist in selecting the optimal dose for future studies.

The company anticipates dosing the first subject early next year, with the study’s completion expected in 2026. An interim analysis of data from PiZZ genotype participants is projected for H2 2025.

Korro Bio chief medical officer Kemi Olugemo said: “Multiple dose treatment with KRRO-110 in a human transgenic mouse model of PiZZ genotype achieved greater than 60% editing and resulted in secretion of functional M-AAT at therapeutically relevant levels.

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“This approval allows us to demonstrate the potential of KRRO-110 in patients with the PiZZ genotype who remain at risk for serious lung and liver complications despite receiving standard-of-care treatment.”

A genetic disorder, AATD is predominantly caused by a mutation in the SERPINA1 gene, leading to pulmonary emphysema and hepatic cirrhosis.

Developed from Korro’s Oligonucleotide Promoted Editing of RNA (OPERA) platform, KRRO-110 utilises the body’s own adenosine deaminase acting on RNA (ADAR) enzyme to edit the SERPINA1 RNA and correct the disease-causing mutation.

Earlier in the month, the company announced its submission for the Phase I/II trial of KRRO-110 to the Bellberry HREC. Additionally, it formed a Clinical Advisory Board (CAB) of lung and liver disease experts to guide the therapy’s clinical development.