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William Blair initiated coverage on Korro Bio Inc. (NASDAQ:KRRO), a private RNA editing company that went public via a reverse merger with Frequency Therapeutics Inc. in an all-stock merger transaction.
The company’s proprietary Oligonucleotide Promoted Editing of RNA (OPERA) platform harnesses the body’s natural base editing system – specifically ADAR (Adenosine Deaminase Acting on RNA) – to make targeted edits to a single RNA base.
William Blair notes that 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.
Also Read: Why Newly Listed Korro Bio Shares Are Gaining Today?
William Blair emphasizes that Wave Life Sciences Ltd (NASDAQ:WVE) is set to release the first clinical data for an ADAR-recruiting therapy in 2024. The analyst initiates with an Outperform rating.
The data from the Phase 1b/2a RestorAATion-2 trial of WVE-006 for severe Alpha-1 antitrypsin deficiency (A1ATD) will be crucial in determining whether the ADAR-mediated RNA editing mechanism is consistent across species—a key concern for investors due to differences in genetic homology.
Korro’s lead asset, KRRO-110, is a lipid nanoparticle (LNP)- encapsulated oligonucleotide designed to correct the E342K PiZ mutation in SERPINA1, which can lead to severe forms of alpha-1 antitrypsin deficiency (A1ATD).
The asset is expected to enter a Phase 1/2 trial in adult A1ATD patients in the second half of 2024, with interim proof-of-concept clinical data in 2025 and trial completion in 2026.
“We believe Korro’s preclinical data package, particularly in the NSG PiZ mouse model, is best-in-class for the ADAR field,” William Blair analyst writes.
“We also believe that Wave’s WVE-006 (GalNAc-conjugated ADAR-recruiting ASO) and Beam Therapeutics Inc’s (NASDAQ:BEAM) BEAM-302 (base editing approach) clinical programs validate Korro’s approach but still leave clear room for differentiation on efficacy or specificity/safety,” the analyst adds.
Price Action: KRRO stock is up 8.64% at $39.50 at the last check on Wednesday.
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