Korro Unveils New Preclinical Data for KRRO-110 at ATS 2024 Conference

Korro Bio, Inc., a biopharmaceutical company listed on Nasdaq as KRRO, is pioneering new genetic medicines for both rare and common diseases. Recently, they revealed significant preclinical data showcasing the efficiency and durability of their candidate KRRO-110 for treating Alpha-1 Antitrypsin Deficiency (AATD).

KRRO-110 has shown a sustained editing efficiency of around 60% and has increased total alpha-1 antitrypsin (AAT) protein levels to over 60µM at the 13-week mark in an in vivo genetic AATD mouse model. Specifically, KRRO-110 demonstrated 35µM of M-AAT one week post-initial dose, with a baseline of 0µM, rising to 45µM of M-AAT by week 13. These results indicate the therapeutic potential of KRRO-110 to restore AAT protein levels effectively.

The data, presented on May 20, 2024, at the American Thoracic Society (ATS) 2024 International Conference in San Diego, also highlighted the utility of a non-human primate surrogate test article. This supports the species translation towards human dose projections, which is a crucial step in drug development. The presentation took place from 11:30 AM to 1:15 PM PT under the abstract number P1299.

Dr. Steve Colletti, Chief Scientific Officer at Korro, remarked on the promising nature of the data, stating that KRRO-110 could surpass the minimum threshold necessary to impact patients positively. He emphasized the potential of KRRO-110 as a leading therapeutic option for AATD.

Korro is on schedule to file a regulatory submission for the first human study of KRRO-110 in AATD patients in the latter half of 2024. This planned study marks a significant milestone in the company's journey towards addressing the unmet medical needs of AATD patients.

Alpha-1 Antitrypsin Deficiency is a genetic disorder caused predominantly by a single nucleotide variant mutation in the SERPINA1 gene, with the "PiZ" mutation being the most common. Approximately 95% of severe cases are homozygous for the PiZ mutation, known as the PiZZ genotype. This disorder can lead to severe progressive lung diseases such as emphysema and chronic obstructive pulmonary disease (COPD), as well as liver disease. There are an estimated 3.4 million people worldwide with different deficiency allele combinations. Currently, the only FDA-approved treatment for AATD in lung disease is augmentation therapy, which involves weekly infusions of pooled human plasma-derived AAT protein. However, this treatment does not fully address the manifestations of AATD.

Korro Bio's approach leverages their proprietary RNA editing platform to develop a new class of genetic medicines. By focusing on RNA editing rather than DNA, their technology aims to deliver more precise and temporary single base edits, potentially offering greater specificity and improved long-term tolerability. Korro's strategy involves using an oligonucleotide-based approach to bring their medicines to patients, supported by their platform's delivery modalities, manufacturing expertise, and established regulatory pathways for approved oligonucleotide drugs.

Based in Cambridge, Massachusetts, Korro continues to advance its portfolio of genetic medicines, aiming to address both rare and highly prevalent diseases.