CAMBRIDGE, Mass. — Korro Bio, Inc., a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases, has nominated its first development candidate, KRRO-110, for the potential treatment of AATD. KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant. KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT. Preclinical development of KRRO-110 is ongoing in preparation for a potential regulatory filing in the second half of 2024.
“The advancement of KRRO-110 highlights the power of our RNA editing platform, OPERA, and we are excited to see what we expect to be the first of many candidates move into the clinic over the coming years,” said Ram Aiyar, PhD, Chief Executive Officer of Korro. “Selecting a development candidate for our AATD program is an important milestone for Korro, but more importantly, for patients. We are confident that the preclinical profile demonstrated by KRRO-110 will make it a potentially game-changing therapeutic candidate that reduces the disease burden and addresses the continued unmet need faced by patients with both liver and lung manifestations of AATD.”
AATD is an inherited, autosomal recessive genetic disorder that is most frequently caused by a SNV mutation in the SERPINA1 gene, the most common of which is the “PiZ” mutation. Greater than 95% of severe clinical cases are homozygous for the PiZ mutation (known as the PiZZ genotype). Korro has generated compelling preclinical data demonstrating high editing efficiency (>50% editing), which it believes is a key threshold to achieving clinically meaningful secretion of normal A1AT protein in an in vivo mouse model, and targeted durability. In addition, Korro has also shown using surrogates that its product candidates have high translation of RNA editing efficiency from mice to non-human primates (NHPs), demonstrating the potential applicability of its approach in humans.
“AATD patients face a critical unmet need given the limited effectiveness of current standard-of-care options for either the liver or the lung manifestations of the disease,” said Dr. Jeffrey Teckman, an expert in AATD from St. Louis University School of Medicine and a long-time advisor to Korro. “Korro’s RNA editing approach has the potential to be transformative for AATD patients and Korro is well-positioned to deliver a best-in-class therapeutic. I look forward to having access to this therapy as quickly as possible.”
About AATD
AATD is an inherited genetic disorder caused by SNVs in the SERPINA1 gene that can lead to severe progressive lung disease, including emphysema and chronic obstructive pulmonary disease (COPD), and liver disease. There are an estimated 3.4 million individuals with deficiency allele combinations worldwide. The only current FDA-approved treatment for AATD is augmentation therapy, a once-weekly infusion of pooled human plasma-derived A1AT protein, which does not adequately address the manifestations of AATD.
About Korro
Korro is a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases using its proprietary RNA editing platform. Korro is generating a portfolio of differentiated programs that are designed to harness the body’s natural RNA editing process to effect a precise yet transient single base edit. By editing RNA instead of DNA, Korro is expanding the reach of genetic medicines by delivering additional precision and tunability, which has the potential for increased specificity and improved long-term tolerability. Using an oligonucleotide-based approach, Korro expects to bring its medicines to patients by leveraging its proprietary platform with precedented delivery modalities, manufacturing know-how, and established regulatory pathways of approved oligonucleotide drugs.
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