William Blair initiated research coverage of Korro Bio, Inc. (KRRO $35.97), a company pioneering ADAR-based RNA-editing therapies for rare diseases with its proprietary OPERA platform.
“Korro is a leader in the adenosine deaminase acting on RNA, or ADAR, editing field, which aims to perform precise adenosine to inosine substitutions to correct single nucleotide polymorphisms or create de novo proteins of novel function with the goal of disease-modifying therapies,” said analyst Myles R. Minter, Ph.D. “The company’s proprietary Oligonucleotide Promoted Editing of RNA, or OPERA, platform combines novel chemistries, computational biology, and delivery systems that recruit endogenous ADAR for RNA editing to selectively correct disease. ADAR editing permits chronic dosing and titratability of a genetic editing medicine without the risk of permanent off-target effects or potential issues with over- or underexpression of target genes.
“We believe Korro’s OPERA platform is uniquely positioned in the genetic medicine space and will be highly disruptive to the RNA editing field,” added Dr. Minter. “With lead asset KRRO-110, a lipid nanoparticle-encapsulated ADAR guide oligonucleotide targeting SERPINA1 mRNA, set to enter the clinic for alpha-1 antitrypsin deficiency, or A1ATD, therapy in the second half of 2024, yield interim data in 2025, and complete trials in 2026, we view this data as not only potentially catalytic for the opportunity in A1ATD but also broadly de-risking for the broader platform. OPERA has the potential power to pivot into tissues outside the liver, for example, CNS neurodegenerative disorders, but also rapidly expand in terms of applications, from genetic correction to protein-protein interaction modulation, and generation of new de novo proteins with altered functions.”
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