CAMBRIDGE, Mass. - Korro Bio, Inc. (NASDAQ: KRRO), a biopharmaceutical company, has presented new preclinical data that suggests its developmental candidate KRRO-110 could be an effective treatment for Alpha-1 Antitrypsin Deficiency (AATD).
The data, unveiled at the American Thoracic Society 2024 International Conference in San Diego, showed that KRRO-110 maintained approximately 60% editing efficiency and achieved greater than 60µM total alpha-1 antitrypsin (AAT) protein levels at 13 weeks in a genetic AATD mouse model.
AATD is a genetic disorder that can lead to severe lung and liver diseases. The current FDA-approved treatment involves weekly infusions of AAT protein, which does not fully address the disease's manifestations. Korro's KRRO-110 aims to restore AAT protein levels to impact patient outcomes positively.
The study demonstrated that KRRO-110 increased levels of M-AAT protein from 0µM at baseline to 35µM one week after the first dose, rising to 45µM at week 13. This sustained response is a promising indication of the candidate's potential efficacy. Additionally, a non-human primate surrogate test article provided data that supports the translation of this treatment to human dose projections.
Steve Colletti, Ph.D., Chief Scientific Officer of Korro, commented on the significance of the data, suggesting that KRRO-110 has the potential to be a best-in-class therapeutic for AATD. The company is on track to submit a regulatory filing for a first-in-human study of KRRO-110 in the second half of 2024.
Korro Bio is leveraging its proprietary RNA editing platform to develop genetic medicines for both rare and prevalent diseases. The platform aims to achieve precise and transient single base edits in RNA, potentially offering increased specificity and improved tolerability over long-term use.
The information reported is based on a press release statement and reflects the data presented at the ATS 2024 International Conference. It is a continuation of Korro's efforts to advance its portfolio of genetic medicines, with KRRO-110 being a key focus in the treatment of AATD.
InvestingPro Insights
As Korro Bio, Inc. (NASDAQ: KRRO) makes strides in the development of its genetic medicines, investors may be curious about the company's financial health and market performance. According to InvestingPro data, Korro Bio has a market capitalization of $504.11 million, reflecting investors' current valuation of the company.
Despite the promising preclinical data for KRRO-110, the company's P/E ratio stands at -2.31, indicating that it is not currently profitable. This is further evidenced by an adjusted P/E ratio for the last twelve months as of Q1 2024 at -6.15.
Investors should note that the company's stock has experienced a significant return over the last week, with a price total return of 8.8%. This may be reflective of positive reactions to the recent preclinical data announcements. Still, it is important to consider the broader picture, as the stock has fared poorly over the last month with a price total return of -18.1%. This volatility could be a point of consideration for potential investors.
Turning to InvestingPro Tips, two key insights emerge. Firstly, analysts have recently revised their earnings upwards for the upcoming period, signaling potential optimism about the company's future performance. Secondly, despite the company holding more cash than debt on its balance sheet, it is quickly burning through cash, which could raise concerns about its long-term financial sustainability. It is worth noting that there are 14 additional tips available on InvestingPro that could further guide investment decisions.
For investors interested in a deeper dive into Korro Bio's financials and market prospects, they can access further insights and tips by visiting InvestingPro. Remember to use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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