Korro Files for KRRO-110 Human Study, Forms Clinical Advisory Board

Korro Bio, Inc., a biopharmaceutical company from Cambridge, Massachusetts, has made significant strides in its efforts to combat Alpha-1 Antitrypsin Deficiency (AATD) through innovative RNA editing technologies. The company announced on November 4, 2024, that it has submitted a regulatory filing to the Bellberry Human Research Ethics Committee (HREC) in Australia for a Phase 1/2 clinical study of its product candidate, KRRO-110. This submission marks a crucial step in Korro's journey toward becoming a clinical-stage company.

Dr. Kemi Olugemo, Chief Medical Officer at Korro, highlighted the importance of this milestone, emphasizing the substantial unmet medical needs of individuals with AATD. Preclinical data suggests that KRRO-110 could potentially offer a best-in-class therapeutic solution due to its unique RNA editing approach. Dr. Olugemo also underscored the invaluable expertise of the assembled Clinical Advisory Board (CAB), which includes leading lung and liver specialists in the field of AATD. This board will play a pivotal role in guiding Korro's clinical and regulatory strategies, ensuring a comprehensive approach to patient care.

Pending approval from the HREC and the acceptance of the clinical trial notification (CTN) by Australia's Therapeutic Goods Administration (TGA), Korro plans to commence dosing the first participant in the Phase 1/2 study in early 2025. An interim data readout is expected in the latter half of 2025, with the study anticipated to conclude in 2026.

Korro’s CEO and President, Dr. Ram Aiyar, expressed pride in the company's progress, recalling the initial nomination of KRRO-110 for AATD in December 2023. He praised the entire Korro team for their contributions toward reaching this point. Dr. Aiyar highlighted the transformative potential of Korro’s proprietary OPERA™ platform, which underpins a robust pipeline of both fully owned and partnered programs. With a solid financial foundation, Korro is well-positioned to complete the Phase 1/2 study and advance subsequent programs.

AATD is a genetic disorder resulting from a specific missense mutation in the SERPINA1 gene. This mutation leads to conditions such as pulmonary emphysema and hepatic cirrhosis in affected adults. KRRO-110, derived from Korro’s novel RNA editing platform, OPERA™, is designed to exploit an enzyme known as Adenosine Deaminase Acting on RNA (ADAR). By editing the “A” variant on the SERPINA1 RNA, KRRO-110 aims to repair the amino acid codon and restore the secretion of normal AAT protein. This process has the potential to clear protein aggregates within liver cells, thereby enhancing liver function and preserving lung function by ensuring adequate levels of normal AAT protein.

Korro Bio is at the forefront of developing a new class of genetic medicines, utilizing its proprietary RNA editing platform to address both rare and prevalent diseases. By focusing on RNA rather than DNA, Korro aims to deliver precise, transient single base edits, enhancing the specificity and long-term tolerability of genetic treatments. The company's approach leverages oligonucleotide-based methods, utilizing established delivery and manufacturing processes, and follows regulatory pathways proven by approved oligonucleotide drugs.

As Korro moves forward with its clinical ambitions, the collaboration with leading experts and a commitment to innovative scientific solutions positions the company to make significant contributions to the treatment of genetic disorders like AATD.