Gene Editing Korro Bio's Preclinical Data 'Best-In-Class', Analyst Initiates With Outperform Rating

William Blair initiated coverage on Korro Bio Inc. 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 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 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.

Read Next:

• Acelyrin Cuts Workforce, Deprioritizes Former Lead Drug Izokibep.

Don’t miss the opportunity to dominate in a volatile market at the Benzinga SmallCAP Conference on Oct. 9-10 at the Chicago Marriott Downtown Magnificent Mile. Get exclusive access to CEO presentations, 1:1 meetings with investors, and valuable insights from top financial experts. Whether you’re a trader, entrepreneur, or investor, this event offers unparalleled opportunities to grow your portfolio and network with industry leaders. Secure your spot and get your tickets today!

Image via Shutterstock