Korro Gets Australian Approval to Start KRRO-110 Clinical Study for Alpha-1 Antitrypsin Deficiency

Korro Bio, Inc., a biopharmaceutical company known for its work on RNA editing for genetic diseases, has announced that it received approval from the Australian Bellberry Human Research Ethics Committee (HREC) and clearance from the Australian Therapeutic Goods Administration (TGA). These approvals will allow Korro to initiate a Phase 1/2a clinical trial of KRRO-110, a treatment for Alpha-1 Antitrypsin Deficiency (AATD).

Dr. Kemi Olugemo, Chief Medical Officer at Korro, expressed excitement about the approval, stating that the treatment had shown promising results in transgenic mouse models. Specifically, a multiple dose treatment with KRRO-110 achieved over 60% editing and resulted in the secretion of functional M-AAT at therapeutically relevant levels. This outcome supports the potential of KRRO-110 to benefit PiZZ genotype patients who are still at risk of serious lung and liver issues despite receiving standard-of-care treatment.

The upcoming study, named REWRITE, is structured into two parts involving single and multiple dose-escalation phases. It will evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including healthy adults and clinically stable AATD patients with the PiZZ genotype. Secondary and exploratory endpoints will include pharmacokinetic and pharmacodynamic parameters to guide optimal dose selection for subsequent studies.

Korro plans to commence dosing the first participant in the first quarter of 2025. An interim readout is expected in the second half of 2025, with the complete study projected to conclude in 2026.

Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder primarily caused by a single missense mutation in the SERPINA1 gene. Individuals with this condition can suffer from pulmonary emphysema and hepatic cirrhosis, among other organ complications. KRRO-110, the first RNA editing oligonucleotide product from Korro's proprietary platform, is designed to correct the genetic defect by editing the RNA. This edit aims to repair an amino acid codon and restore the secretion of normal Alpha-1 Antitrypsin (AAT) protein. By repairing the protein, KRRO-110 could potentially clear harmful protein aggregates in liver cells and improve both liver and lung function.

Korro Bio specializes in developing genetic medicines for both rare and common diseases through RNA editing. Its proprietary RNA editing platform aims to harness the body’s natural RNA editing process to make precise yet transient edits. This approach is considered to offer greater precision and long-term tolerability compared to DNA editing. Korro’s strategy involves using oligonucleotide-based methods and leveraging established regulatory pathways for approved drugs to bring its treatments to patients.

The company is based in Cambridge, Massachusetts, and is dedicated to advancing its portfolio of genetic medicines through rigorous clinical development and strategic regulatory planning.