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Korro Announces Selection of its First Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
KRRO-110 is the first RNA editing oligonucleotide candidate from Korro’s proprietary OPERA™ platform Korro has demonstrated the ability to repair the
About this update from Korro Bio, Inc.
[{"type":"text","content":"KRRO-110 is the first RNA editing oligonucleotide candidate from Korro’s proprietary OPERA™ platform Korro has demonstrated the ability to repair the misfolding of the Alpha-1 Antitrypsin (A1AT) protein containing the Z mutation, as well as reduce liver aggregates, in a preclinical mouse modelKorro has also demonstrated high editing efficiency (>50% editing) in a preclinical mouse model, leading to increased secretion of normal A1AT protein to levels that Korro believes has the potential to be clinically meaningful for AATD patients Korro expects regulatory filing in the second half of 2024 CAMBRIDGE, Mass., Dec. 07, 2023 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), 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 d...