Core Insights - Korro Bio, Inc. is advancing its development candidate KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD), showcasing promising preclinical data [1][3] - The company is on track to submit a regulatory filing for a first-in-human study of KRRO-110 in AATD patients in the second half of 2024 [3] Preclinical Data - KRRO-110 demonstrated approximately 60% editing efficiency and increased total AAT protein levels to greater than 60µM at week 13, with previous data showing efficacy up to week 9 [2][6] - The study included a non-human primate surrogate test article, supporting species translation towards human dose projections [2] AATD Overview - AATD is an inherited genetic disorder primarily caused by a mutation in the SERPINA1 gene, with over 95% of severe cases being homozygous for the PiZ mutation [4] - The condition can lead to severe lung and liver diseases, affecting an estimated 3.4 million individuals globally [4] Company Background - Korro Bio focuses on developing a new class of genetic medicines using its proprietary RNA editing platform, aiming to provide precise and transient single base edits [5] - The company is based in Cambridge, Massachusetts, and is leveraging established regulatory pathways for oligonucleotide drugs to bring its medicines to patients [5]
Korro Presents Additional Preclinical Data for KRRO-110 at the American Thoracic Society 2024 International Conference