Gene Editing Korro Bio's Preclinical Data 'Best-In-Class', Analyst Initiates With Outperform Rating
Gene Editing Korro Bio's Preclinical Data 'Best-In-Class', Analyst Initiates With Outperform Rating
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.
William Blair开始覆盖Korro Bio Inc. (纳斯达克：KRRO)——一家通过与Frequency Therapeutics Inc.进行全股票交易逆向并购而上市的私人RNA编辑公司。
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.
该公司的专有Oligonucleotide Promoted Editing of RNA (OPERA) 平台利用人体天然的基因编辑系统，尤其是ADAR (Adenosine Deaminase Acting on RNA)——来针对单一RNA碱基进行有针对性的编辑。
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.
William Blair指出，ADAR编辑能够实现基因编辑药物的慢性给药和可调节性，而又不会存在永久性的离靶影响，同时也不会有过/低表达的靶基因存在潜在问题。
Also Read: Why Newly Listed Korro Bio Shares Are Gaining Today?
同时阅读：为什么Korro Bio的新上市股票今天涨了？
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.
William Blair强调Wave Life Sciences Ltd (纳斯达克：WVE)计划在2024年发布第一批招募ADAR疗法的临床数据。分析师给予”跑赢大盘“评级。
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.
来自WVE-006严重α1抗胰蛋白酶缺陷（A1ATD）的1b/2a RestorAATion-2试验的数据将至关重要地确定ADAR调节的RNA编辑机制是否跨物种一致——这是投资者的一大关注点，因为不同基因同源性的差异。
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).
Korro的主要资产KRRO-110，是一种脂质纳米粒（LNP）包装的寡核苷酸，旨在纠正SERPINA1中的E34.2万PiZ突变，该缺陷可以导致α1抗胰蛋白酶缺陷（A1ATD）的严重形式。
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.
该项目预计在2024年下半年进入针对成年A1ATD患者的1/2期试验，并于2025年提交中期概念验证临床数据，2026年完成试验。
"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.
William Blair分析师写道，“我们相信Korro在NSG PiZ小鼠模型的预临床数据是ADAR领域最好的。”
"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.
“我们还相信Wave的WVE-006（GalNAc结合的ADAR-招募ASO）和Beam Therapeutics Inc.的BEAm-302（基因编辑方法）的临床项目验证了Korro的方法，但仍然有明显的差异，因为我们期待Korro的疗效或特异性/安全性。” 该分析师补充道。
Price Action: KRRO stock is up 8.64% at $39.50 at the last check on Wednesday.
股价行情：KRRO股票在周三最后一次检查时上涨8.64%，至39.50美元。
- Acelyrin Cuts Workforce, Deprioritizes Former Lead Drug Izokibep.
- Acelyrin削减工作人员，降低原主力药品Izokibep的优先级。
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!
不要错过机会，在10月9-10日的Benzinga SmallCAP会议上主导波动市场。获得CEO演示文稿的独家访问，与投资者进行1：1会议，并获得来自顶尖金融专家的宝贵见解。无论您是交易员、企业家还是投资者，此活动都提供了无与伦比的机会，使您的投资组合得到增长，并与行业领袖建立联系。现在就立即预订您的位置并购买门票吧！
Image via Shutterstock
图片来自shutterstock。