Korro Bio Methodology - Korro Bio利用机器学习优化寡核苷酸，并结合对ADAR生物学的深入理解、定制的递送方式和专业的寡核苷酸化学技术，实现RNA编辑[7] - Korro Bio采用成熟和新型的化学修饰来改善ADAR引导寡核苷酸的药理学特性[10] Machine Learning for Chemically Modified Oligonucleotide Design - 针对单一靶点，在20%的体外数据上测试模型，预测结果与体外编辑结果相差在7%以内[29] - 通过迭代设计批次，机器学习能够促进mRNA编辑[33] - 增加化学特征的提取，会导致模型总体误差增加约1%[47] - 化学广义图神经网络（GNN）可以帮助公司进行新的修饰滴定[63] - GNN模型在序列上的r值为0.06，而经过化学修饰的GNN模型，r值可以达到0.72[64] Methods for Increasing Use-Cases for ML Oligonucleotide Design - 序列单体模型的r值为0.78，原子模型的r值为0.66，而序列模型的r值为0.80[48] - 在寡核苷酸中，化学特征可以改善8种未见过的修饰的总体误差[56] - 靶标不可知的特征提取方法，通过插入与靶标的关系，可以用于新的靶标[69, 70] - 寡核苷酸-靶标相互作用特征可以为新的靶标和细胞系带来更好的模型[71] - ACTB在Target Agnostic的情况下ρ = -0.07，在使用Sequence Model Predictions的情况下ρ = 0.61[72]

Summary of Cora Bio Conference Call Company Overview - **Company**: Cora Bio - **Industry**: Biotechnology, specifically focusing on genetic medicines for chronic diseases [1][2] Core Points and Arguments - **Mission and Strategy**: Cora Bio aims to develop genetic medicines targeting complex chronic diseases by leveraging pharmacology and genetic insights. The focus is on creating a toolbox for biological pathway activation that is not available with small molecules [3][4] - **RNA Editing Technology**: The company utilizes RNA editing through oligonucleotides to modify specific genes, aiming for high levels of editing (above 50%) to restore normal protein function in patients [4][5] - **Pipeline Development**: Cora Bio has a three:two:one strategy, planning to advance three candidates into clinical trials across two tissue types. The third indication is expected to target the central nervous system (CNS) for ALS [6][7] - **Regulatory Environment**: The company has engaged with the FDA, receiving orphan drug designation and conducting pre-IND meetings. There is concern about the increasing regulatory bar but confidence in the data driving approval processes [10][12][13] - **Preclinical Data**: Cora Bio presented promising preclinical data showing significant editing efficiency (60-65%) in animal models, which translates well to protein levels in circulation [18][20] - **Lipid Nanoparticle Delivery**: The company uses lipid nanoparticles for drug delivery, which have shown safety in patients with liver conditions. The focus is on optimizing dosing to achieve therapeutic protein levels [21][23][25] Important but Overlooked Content - **Patient Population Insights**: The company discussed the baseline liver function levels in patients with ZZ mutations, indicating that many have normal liver function, which may mitigate concerns about toxicity from lipid nanoparticles [22][23] - **Dosing Strategy**: Cora Bio aims for a dosing frequency of every three to six weeks, with the goal of achieving therapeutic protein levels that are comparable to normal individuals [26] - **Regulatory Pathway Confidence**: The company believes that demonstrating protein levels close to normal and achieving significant editing levels could support discussions for accelerated approval with regulators [39][40] - **Financial Position**: Cora Bio has a cash runway into 2027, with $139 million on the balance sheet and potential milestones from collaborations, indicating a solid financial foundation for ongoing development [47]

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The company reported decreased total assets, increased net loss, and initial collaboration revenue in Q1 2025 [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to $202.2 million as of March 31, 2025, with a corresponding decrease in total stockholders' equity Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $64,144 | $55,643 | | Total current assets | $121,235 | $131,532 | | Total assets | $202,207 | $226,240 | | Total current liabilities | $12,881 | $14,960 | | Total liabilities | $63,250 | $65,825 | | Total stockholders' equity | $138,957 | $160,415 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Q1 2025 saw the first collaboration revenue of $2.6 million, but net loss increased to $23.4 million due to higher R&D expenses Q1 2025 vs Q1 2024 Statement of Operations (in thousands, except per share data) | Metric | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | | :--- | :--- | :--- | | Collaboration revenue | $2,550 | $0 | | Research and development expenses | $19,739 | $13,572 | | General and administrative expenses | $7,831 | $7,881 | | Loss from operations | ($25,020) | ($21,453) | | Net loss | ($23,387) | ($19,557) | | Net loss per share, basic and diluted | ($2.49) | ($2.44) | [Condensed Consolidated Statements of Stockholders' Equity](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders%27%20Equity) Total stockholders' equity decreased to $139.0 million by March 31, 2025, primarily due to the $23.4 million net loss - Total stockholders' equity decreased by **$21.4 million** during the first quarter of 2025, mainly due to the net loss of **$23.4 million**[23](index=23&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities was $24.5 million, offset by $32.8 million from investing activities, resulting in an $8.5 million net cash increase Q1 2025 vs Q1 2024 Cash Flow Summary (in thousands) | Activity | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($24,452) | ($21,891) | | Net cash provided by (used in) investing activities | $32,781 | ($5,611) | | Net cash provided by financing activities | $172 | $139 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail cash runway, collaboration revenue from Novo Nordisk, a Genevant milestone, and a subsequent 20% workforce reduction - The company expects its cash, cash equivalents, and marketable securities of **$139.0 million** as of March 31, 2025, will be sufficient to fund operations for at least **12 months** from the financial statement issuance date[31](index=31&type=chunk) - In January 2025, the company triggered a **$1.5 million** development milestone payment to Genevant Sciences GmbH after dosing the first participants in its Phase 1/2a REWRITE clinical trial for KRRO-110[63](index=63&type=chunk) - Under the Novo Nordisk agreement, the company recognized **$2.6 million** in collaboration revenue for the three months ended March 31, 2025. The total transaction price for the first program target is estimated at **$40.1 million**[68](index=68&type=chunk) - On May 7, 2025, the company initiated a strategic plan including a workforce reduction of approximately **20%**, with estimated one-time restructuring charges of about **$1.2 million**[86](index=86&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's RNA editing focus, increased Q1 2025 net loss due to R&D, and a cash runway into 2027 [Overview](index=23&type=section&id=Overview) Korro Bio, a clinical-stage RNA editing company, initiated a Phase 1/2a trial for KRRO-110 and announced a 20% workforce reduction - The company is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on RNA editing[88](index=88&type=chunk) - The lead clinical program, KRRO-110 for AATD, began its Phase 1/2a REWRITE clinical trial in January 2025. Interim data from healthy volunteers and AATD patients is expected in the second half of 2025[91](index=91&type=chunk) - In May 2025, the company announced a strategic plan to streamline operations, including a workforce reduction of approximately **20%**, with expected restructuring charges of **$1.2 million**[92](index=92&type=chunk) - The company has incurred significant operating losses since inception, with a net loss of **$23.4 million** for Q1 2025 and an accumulated deficit of **$290.0 million** as of March 31, 2025[95](index=95&type=chunk) [Results of Operations](index=28&type=section&id=Results%20of%20Operations) Q1 2025 saw $2.6 million in collaboration revenue, while R&D expenses increased by $6.2 million, primarily for the KRRO-110 trial R&D Expense Breakdown (in thousands) | Expense Category | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | KRRO-110 (AATD) external expenses | $6,122 | $2,249 | $3,873 | | Other research and pre-development | $4,350 | $3,300 | $1,050 | | Personnel expenses | $6,440 | $5,213 | $1,227 | | Facilities expenses | $2,827 | $2,810 | $17 | | **Total R&D expenses** | **$19,739** | **$13,572** | **$6,167** | - The increase in R&D expenses was primarily due to a **$3.9 million** increase in KRRO-110 external costs, including a **$2.4 million** increase in clinical trial activities and a **$1.5 million** development milestone payment to Genevant[110](index=110&type=chunk) - General and administrative expenses were largely unchanged at **$7.8 million** for Q1 2025 compared to **$7.9 million** in Q1 2024[112](index=112&type=chunk) [Liquidity and Capital Resources](index=29&type=section&id=Liquidity%20and%20Capital%20Resources) The company held $139.0 million in cash and marketable securities, expected to fund operations into 2027, despite $24.5 million in Q1 operating cash use - As of March 31, 2025, the company had cash, cash equivalents and marketable securities of **$139.0 million**[114](index=114&type=chunk) - The company expects its cash position as of March 31, 2025, will be sufficient to fund operating expenses and capital expenditure requirements into **2027**[117](index=117&type=chunk) Cash Flow Summary (in thousands) | Activity | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($24,452) | ($21,891) | | Net cash provided by (used in) investing activities | $32,781 | ($5,611) | | Net cash provided by financing activities | $172 | $139 | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=33&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risk is primarily interest rate sensitivity on investments, with minimal foreign currency exposure - The company's cash, cash equivalents, and marketable securities of **$139.0 million** are sensitive to changes in interest rates, but a **10%** change is not expected to have a material effect on their fair market value[131](index=131&type=chunk) - The company has minimal exposure to foreign currency risk as most contracts with vendors are denominated in U.S. dollars[132](index=132&type=chunk) [Item 4. Controls and Procedures](index=33&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal controls - The CEO and CFO concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level as of March 31, 2025[134](index=134&type=chunk) - No material changes to the company's internal control over financial reporting occurred during the first quarter of 2025[135](index=135&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=34&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently party to any legal proceedings expected to have a material adverse effect on its business - As of the date of the report, the company is not party to any claim or litigation that is expected to have a material adverse effect on its business[137](index=137&type=chunk) [Item 1A. Risk Factors](index=34&type=section&id=Item%201A.%20Risk%20Factors) The company faces extensive risks including financial losses, unproven RNA editing technology, clinical development challenges, third-party reliance, and intellectual property issues [Risks Related to Our Business](index=34&type=section&id=Risks%20Related%20to%20Our%20Business) The company faces risks from its history of significant losses, need for substantial funding, and early-stage clinical development of KRRO-110 - The company has incurred significant losses since inception, with an accumulated deficit of **$290.0 million** as of March 31, 2025, and expects to incur losses for the foreseeable future[139](index=139&type=chunk) - The company will require substantial additional funding and may be forced to delay, reduce, or eliminate research programs if unable to raise capital when needed[145](index=145&type=chunk) - The company has only dosed a few participants in its Phase 1/2a trial of KRRO-110 and has not completed any clinical trials. Favorable preclinical results are not predictive of future clinical trial outcomes[148](index=148&type=chunk) [Risks Related to Discovery, Development and Commercialization](index=41&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%20and%20Commercialization) Risks include unproven RNA editing technology, potential clinical trial failures, complex manufacturing, reliance on third-party LNP, and intense market competition - RNA editing is a novel technology with limited clinical validation for human therapeutic use, and the company's approaches are unproven and may never lead to marketable products[166](index=166&type=chunk) - The process of manufacturing oligonucleotides is complex and presents risks in process development and scaling up, which could be affected by cost-overruns, delays, and quality control issues[182](index=182&type=chunk)[183](index=183&type=chunk) - The company licenses the LNP delivery technology for KRRO-110 from Genevant, and termination of this agreement could delay or terminate development of the product candidate[181](index=181&type=chunk) - The pharmaceutical industry is intensely competitive, and the company faces competition from large companies with greater resources and from new drugs that may be more effective, safer, or less expensive[199](index=199&type=chunk)[200](index=200&type=chunk) [Risks Related to Regulatory, Legal, and Clinical Trials](index=59&type=section&id=Risks%20Related%20to%20Regulatory%2C%20Legal%2C%20and%20Clinical%20Trials) The company faces regulatory uncertainty for oligonucleotides, potential legislative impacts, extensive post-marketing requirements, and compliance risks with healthcare and data privacy laws - Developing oligonucleotides, a relatively new class of drugs, carries increased risk as the FDA and other regulators have limited experience, which may lead to a complex, uncertain, and lengthy review process[211](index=211&type=chunk) - The company faces risks from enacted and future legislation, such as the Affordable Care Act (ACA), which may increase the difficulty and cost of obtaining marketing approval and affect product pricing[212](index=212&type=chunk)[214](index=214&type=chunk) - Any approved product will be subject to extensive post-marketing regulatory requirements, and failure to comply can result in restrictions, withdrawal from the market, or penalties[229](index=229&type=chunk) - The company is subject to a variety of privacy and data security laws, such as GDPR, and failure to comply could result in significant fines and harm to the business[265](index=265&type=chunk) [Risks Related to Our Third Party Relationships](index=78&type=section&id=Risks%20Related%20to%20Our%20Third%20Party%20Relationships) The company heavily relies on third parties for clinical trials and manufacturing, faces risks from their performance, and depends on collaborator efforts like Novo Nordisk - The company relies on third parties like CROs and clinical investigators to conduct clinical trials and research, and these parties may not perform satisfactorily or meet deadlines[268](index=268&type=chunk) - The company relies on third parties for the supply and manufacture of materials for research, preclinical, and clinical activities, and may be unable to identify or switch to alternative manufacturers on acceptable terms[277](index=277&type=chunk)[278](index=278&type=chunk) - The success of the collaboration with Novo Nordisk and any future partnerships depends heavily on the efforts and resources of the collaborators, which are outside the company's direct control[272](index=272&type=chunk) [Risks Related to Our Personnel, Operations and Growth](index=85&type=section&id=Risks%20Related%20to%20Our%20Personnel%2C%20Operations%20and%20Growth) Success depends on retaining key personnel, with the recent workforce reduction potentially hindering recruitment and future growth management - The company is highly dependent on its senior management and key scientific personnel, and the loss of their services could delay R&D programs and harm the business[286](index=286&type=chunk) - The recent workforce reduction in May 2025 could impede future efforts to recruit and hire qualified personnel needed for operational expansion[286](index=286&type=chunk)[287](index=287&type=chunk) - The company may encounter difficulties managing future growth as it expands its R&D, manufacturing, and commercialization capabilities, which could disrupt operations[287](index=287&type=chunk) [Risks Related to Intellectual Property](index=87&type=section&id=Risks%20Related%20to%20Intellectual%20Property) Success depends on protecting intellectual property, facing risks of patent invalidation, infringement lawsuits, and challenges from evolving patent laws - The company's success depends on its ability to obtain and maintain patents and other intellectual property rights, but it cannot be certain that its patent applications will issue or that issued patents will be enforceable or broad enough to prevent competition[289](index=289&type=chunk)[292](index=292&type=chunk) - The company may be involved in lawsuits to protect its patents or defend against third-party claims of infringement, which are expensive, time-consuming, and have uncertain outcomes[305](index=305&type=chunk)[312](index=312&type=chunk) - Changes in U.S. patent law, such as the America Invents Act, and rulings by the Supreme Court have increased uncertainty regarding patent protection and enforcement[343](index=343&type=chunk)[345](index=345&type=chunk) - The company relies on trade secrets and confidentiality agreements to protect proprietary information, but these measures may be breached or may not provide adequate protection[348](index=348&type=chunk)[349](index=349&type=chunk) [General Risk Factors](index=109&type=section&id=General%20Risk%20Factors) General risks include stock price volatility, high public company costs, anti-takeover provisions, significant stockholder control, and macroeconomic uncertainties affecting NOLs - The company's stock price is volatile and can be affected by numerous factors, including clinical trial results, competitor announcements, and general market conditions[367](index=367&type=chunk) - Executive officers, directors, and principal stockholders beneficially own approximately **71%** of the company's common stock, giving them significant influence over stockholder matters[384](index=384&type=chunk) - The company's ability to use its net operating loss (NOL) carryforwards of **$352.3 million** (federal) and **$319.7 million** (state) may be limited by 'ownership change' rules under Section 382 of the tax code[390](index=390&type=chunk)[391](index=391&type=chunk) - The business may be adversely affected by macroeconomic conditions, including inflation, rising interest rates, and volatile market conditions[393](index=393&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=122&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section is not applicable for the current reporting period - Not applicable[395](index=395&type=chunk) [Item 5. Other Information](index=122&type=section&id=Item%205.%20Other%20Information) No new Rule 10b5-1(c) trading plans were adopted by officers or directors during Q1 2025 - During the three months ended March 31, 2025, no officers or directors adopted new trading plans for the company's securities under Rule 10b5-1(c)[398](index=398&type=chunk) [Item 6. Exhibits](index=123&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including corporate governance documents and officer certifications - The exhibits filed include corporate governance documents, an employment agreement for Loïc Vincent, and required officer certifications under the Sarbanes-Oxley Act[400](index=400&type=chunk)

[Business and Operational Highlights](index=1&type=section&id=Business%20and%20Operational%20Highlights) Korro implements a 20% workforce reduction to extend its cash runway into 2027, focusing on KRRO-110 clinical trials and Novo Nordisk collaboration, with $139.0 million cash at Q1 2025 - The company is prioritizing the advancement of its Phase 1/2a REWRITE clinical trial for KRRO-110, with interim data anticipated in the second half of 2025[4](index=4&type=chunk) - Korro is streamlining operations and reducing its workforce by approximately **20%** to extend its cash runway into **2027** and focus on key program milestones[5](index=5&type=chunk)[7](index=7&type=chunk) - The company estimates it will incur approximately **$1.2 million** in one-time restructuring charges, primarily in the second quarter of 2025, related to employee severance and termination costs[5](index=5&type=chunk) Key Operational and Financial Metrics | Metric | Value/Status | | :--- | :--- | | **Cash Position (Q1 2025)** | $139.0 million | | **Cash Runway** | Expected to extend into 2027 | | **KRRO-110 Interim Data** | On track for H2 2025 | | **Second Development Candidate** | Expected to be announced in 2025 | [Pipeline and Clinical Development](index=1&type=section&id=Pipeline%20and%20Clinical%20Development) Korro's KRRO-110 Phase 1/2a REWRITE trial for AATD is advancing with H2 2025 interim data expected, while the company executes its '3-2-1 strategy' and progresses its Novo Nordisk collaboration - The Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD is advancing with expansion into New Zealand and other geographies. Interim data is expected in **H2 2025**, with trial completion in **2026**[6](index=6&type=chunk)[8](index=8&type=chunk)[12](index=12&type=chunk) - KRRO-110 is an RNA editing oligonucleotide designed to repair the SERPINA1 RNA mutation, aiming to restore normal AAT protein, clear liver protein aggregates, and preserve lung function[17](index=17&type=chunk) - Korro is executing its **'3-2-1 strategy'** with the goal of having **three clinical-stage programs** and targeting **two tissue types** by the end of **2027**[12](index=12&type=chunk) - The company plans to nominate a development candidate for a rare metabolic disorder by the end of **2025**[12](index=12&type=chunk) - The collaboration with Novo Nordisk is progressing, with a focus on advancing up to **two preclinical programs** for cardiometabolic diseases using Korro's OPERA™ platform[12](index=12&type=chunk) - The leadership team was strengthened with the appointments of Dr. Loïc Vincent as Chief Scientific Officer and Dr. GaoZhong Zhu as Senior Vice President of Chemistry, Manufacturing and Controls (CMC)[12](index=12&type=chunk) [First Quarter 2025 Financial Results](index=2&type=section&id=First%20Quarter%202025%20Financial%20Results) Korro reported **$2.6 million** in Q1 2025 collaboration revenue, with R&D expenses at **$19.7 million** and a net loss of **$23.4 million**, ending the quarter with **$139.0 million** cash expected to fund operations into **2027** Key Financial Highlights (Q1 2025 vs. Q1 2024) | Financial Metric | Q1 2025 | Q1 2024 | Key Driver | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $2.6M | $0 | Revenue earned from Novo Nordisk agreement | | R&D Expenses | $19.7M | $13.6M | Increase in KRRO-110 external R&D and personnel costs | | G&A Expenses | $7.8M | $7.9M | Remained relatively flat | | Net Loss | $23.4M | $19.6M | Increased operating expenses | - Cash, cash equivalents, and marketable securities were **$139.0 million** as of March 31, 2025, compared to **$163.1 million** as of December 31, 2024[10](index=10&type=chunk) - The current cash position is expected to fund operating expenses and capital expenditure requirements into **2027**[10](index=10&type=chunk) [Financial Statements](index=6&type=section&id=Financial%20Statements) Korro's unaudited condensed consolidated financial statements for Q1 2025 show a net loss of **$23.4 million**, total assets of **$202.2 million**, and a cash position of **$139.0 million** as of March 31, 2025 [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Korro reported a net loss of **$23.4 million** (or **$2.49** per share) for Q1 2025, compared to **$19.6 million** (or **$2.44** per share) in Q1 2024, driven by **$2.6 million** in collaboration revenue and increased R&D expenses Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share data) | Metric (in thousands, except per share data) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Collaboration revenue | $2,550 | $— | | Research and development | $19,739 | $13,572 | | General and administrative | $7,831 | $7,881 | | Loss from operations | $(25,020) | $(21,453) | | **Net loss** | **$(23,387)** | **$(19,557)** | | **Net loss per share, basic and diluted** | **$(2.49)** | **$(2.44)** | | Weighted-average shares outstanding | 9,384,266 | 8,019,626 | [Selected Condensed Consolidated Balance Sheet Data](index=7&type=section&id=Selected%20Condensed%20Consolidated%20Balance%20Sheet%20Data) As of March 31, 2025, Korro's balance sheet shows total assets of **$202.2 million**, total liabilities of **$63.3 million**, and **$139.0 million** in cash, cash equivalents, and marketable securities Selected Condensed Consolidated Balance Sheet Data (in thousands) | Metric (in thousands) | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Cash, cash equivalents and marketable securities** | **$138,992** | **$163,054** | | Working capital | $108,354 | $116,572 | | Total assets | $202,207 | $226,240 | | Total liabilities | $63,250 | $65,825 | | Total stockholders' equity | $138,957 | $160,415 |

Core Insights - Korro Bio, Inc. is focused on developing genetic medicines through RNA editing for both rare and prevalent diseases, with a significant milestone expected in the second half of 2025 regarding the KRRO-110 clinical trial for Alpha-1 Antitrypsin Deficiency (AATD) [2][5][16] Financial Performance - As of March 31, 2025, Korro reported cash, cash equivalents, and marketable securities totaling $139.0 million, down from $163.1 million at the end of 2024, which is expected to fund operations into 2027 [9][23] - Collaboration revenue for the first quarter of 2025 was $2.6 million, a notable increase from zero in the same period of 2024, attributed to the partnership with Novo Nordisk [10] - Research and Development (R&D) expenses rose to $19.7 million in Q1 2025 from $13.6 million in Q1 2024, primarily due to increased costs associated with KRRO-110 [11] - General and Administrative (G&A) expenses slightly decreased to $7.8 million in Q1 2025 from $7.9 million in Q1 2024 [12] - The net loss for Q1 2025 was $23.4 million, compared to a net loss of $19.6 million in Q1 2024 [13] Operational Updates - The company is streamlining operations and reducing its workforce by approximately 20% to focus on key program milestones, including the completion of the Phase 1/2a REWRITE clinical trial in 2026 [3][5] - Korro is executing a 3-2-1 strategy aimed at establishing three clinical-stage development programs targeting two tissue types by leveraging its OPERA platform [6][16] - The leadership team has been strengthened with new appointments, enhancing the company's capabilities in drug discovery and development [6][7] Pipeline Developments - The interim readout from the Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD is on track for the second half of 2025, with trial completion expected in 2026 [5][6][16] - Korro plans to announce a second development candidate by the end of 2025, focusing on a rare metabolic disorder program targeting the liver [16] - The collaboration with Novo Nordisk continues to progress, with efforts to advance up to two programs targeting cardiometabolic diseases [7][16]

Core Insights - Korro Bio, Inc. has appointed Dr. Loïc Vincent as Chief Scientific Officer, bringing over 20 years of drug development experience to the company [2][3] - The company is advancing multiple candidates in its pipeline, including KRRO-110, currently in Phase 1/2a clinical study for Alpha-1 Antitrypsin Deficiency [2] - Korro's RNA-editing platform aims to develop genetic medicines for both rare and prevalent diseases, enhancing precision and long-term tolerability [4] Company Overview - Korro Bio is a clinical-stage biopharmaceutical company focused on RNA editing to create genetic medicines [4] - The company utilizes an oligonucleotide-based approach to expand the reach of genetic therapies, leveraging established regulatory pathways [4] - Korro is headquartered in Cambridge, Massachusetts [4] Leadership Appointments - Dr. GaoZhong Zhu has joined as Senior Vice President of Chemistry, Manufacturing and Controls, with over 25 years of experience in pharmaceutical development [5] - Oliver Dolan has been promoted to Principal Accounting Officer, having served as Senior Vice President of Finance since January 2024 [5]

Financial Performance - The company reported a net loss of $83.6 million for the year ended December 31, 2024, compared to a net loss of $81.2 million for 2023, reflecting an increase in losses of $2.4 million [532]. - The company has an accumulated deficit of $266.6 million as of December 31, 2024, indicating ongoing financial challenges [532]. - The company has incurred an accumulated deficit of $266.6 million as of December 31, 2024, and anticipates continued significant operating losses and negative cash flows [552]. - Net cash used in operating activities was $60.1 million in 2024, compared to $67.3 million in 2023, reflecting a decrease in cash outflow [559][560]. - Total other income, net rose to $8.5 million in 2024 from $3.4 million in 2023, driven by a $5.1 million increase in interest income from cash and marketable securities [550]. Research and Development - Research and development expenses increased to $63.6 million in 2024 from $57.2 million in 2023, primarily due to a $19.3 million rise in external expenses related to the KRRO-110 program [547]. - The KRRO-110 program is currently in a Phase 1/2a clinical trial, with the first participants dosed in January 2025, and interim data expected in the second half of 2025 [530]. - The company expects to continue incurring significant expenses related to its genetic medicine discovery efforts and clinical trials [555]. - The company anticipates significant increases in both research and development and general administrative expenses as it expands its operations and headcount [542]. Revenue and Collaboration - The company recognized $2.3 million in collaboration revenue during the year ended December 31, 2024, from a research collaboration with Novo Nordisk, while no collaboration revenue was recognized in 2023 [545]. - The company has not generated any revenue from product sales and does not expect to do so in the near future, relying on external funding for operations [534]. Operating Expenses - The total operating expenses for 2024 were $94.2 million, up from $84.5 million in 2023, marking an increase of $9.6 million [544]. - General and administrative expenses increased to $30.5 million in 2024 from $27.3 million in 2023, a rise of $3.3 million, primarily due to a $1.9 million increase in IT and other costs, and a $1.1 million increase in professional fees [549]. Cash Flow and Financing - Net cash used in investing activities was $123.3 million in 2024, primarily due to $146.3 million spent on marketable securities [561]. - Net cash provided by financing activities was $69.4 million in 2024, mainly from a private placement of common stock [563]. - The company has raised approximately $223.6 million from the sale of convertible preferred stock and $117.3 million from common stock sales prior to a business combination in November 2023 [531]. - The company entered into a sales agreement allowing for the issuance of up to $100.0 million in common stock through at-the-market offerings, with no shares sold as of December 31, 2024 [553]. Future Outlook - Future capital requirements will depend on various factors, including the progress of clinical trials and regulatory approvals, with potential dilution of stockholder interests if additional capital is raised [556]. - The FDA granted orphan drug designation to KRRO-110 for the treatment of AATD in March 2025, enhancing its development prospects [530]. - Future operations may be adversely affected by inflation [576]. - Inflation has increased costs related to labor, research, manufacturing, and development, but has not materially affected financial statements to date [576]. Currency and Economic Factors - The company has minimal exposure to foreign currency exchange rate fluctuations due to short transaction durations [575].

Financial Performance - Korro reported a net loss of $83.6 million for the year ended December 31, 2024, compared to a net loss of $81.2 million in 2023, reflecting an increase of 2.9%[14] - As of December 31, 2024, Korro had cash, cash equivalents, and marketable securities totaling $163.1 million, down from $166.1 million at the end of 2023[9] Research and Development - Research and Development (R&D) expenses increased to $63.6 million in 2024 from $57.2 million in 2023, representing an increase of 11.2%[11] - The Phase 1/2a REWRITE clinical study of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD) has completed dosing of the first two cohorts, with an interim readout expected in the second half of 2025[6] - Korro aims to establish three clinical-stage development programs targeting two tissue types with a single RNA-editing platform by the end of 2027[12] - Korro plans to announce its second development candidate by the end of 2025, focusing on a rare metabolic disorder program targeting the liver[12] Collaboration and Designation - Collaboration revenue for 2024 was $2.3 million, a significant increase from no collaboration revenue in 2023[10] - The FDA granted Orphan Drug Designation to KRRO-110 for the treatment of AATD, providing various development incentives[8] Leadership and Strategy - The company strengthened its leadership team with key appointments, including the formation of a Clinical Advisory Board[12] - The company expects its cash position to fund operations into the second half of 2026, supporting the KRRO-110 clinical study and additional product candidates[6]

Core Insights - Korro Bio, Inc. is focused on developing genetic medicines through RNA editing for both rare and prevalent diseases, reporting full-year 2024 financial results and progress updates [1][17] Pipeline and Business Updates - The Phase 1/2a REWRITE clinical study of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD) has completed dosing of the first two cohorts, with an interim readout expected in the second half of 2025 [5][6] - KRRO-110 has received Orphan Drug Designation from the FDA for AATD treatment, providing various development incentives [6] - The company is executing a 3-2-1 strategy aiming to establish three clinical-stage development programs targeting two tissue types by the end of 2027, with a second development candidate expected to be announced by the end of 2025 [5][6] - Korro has strengthened its leadership team with key appointments, including the formation of a Clinical Advisory Board [6][7] Financial Performance - As of December 31, 2024, Korro reported cash, cash equivalents, and marketable securities of $163.1 million, down from $166.1 million in 2023, with a cash runway expected to fund operations into the second half of 2026 [9][24] - Collaboration revenue for 2024 was $2.3 million, compared to no collaboration revenue in 2023, attributed to the partnership with Novo Nordisk [10] - Research and Development (R&D) expenses increased to $63.6 million in 2024 from $57.2 million in 2023, driven by external research and development costs related to KRRO-110 [11] - General and Administrative (G&A) expenses rose to $30.5 million in 2024 from $27.3 million in 2023, primarily due to increased IT and professional fees [12] - The net loss for 2024 was $83.6 million, compared to a net loss of $81.2 million in 2023 [13][22] Upcoming Milestones - Interim data from the REWRITE clinical study is expected in the second half of 2025, with study completion anticipated in 2026 [16] - Korro plans to nominate a development candidate for a rare metabolic disorder program targeting the liver by the end of 2025 [16]

Core Insights - Korro Bio, Inc. has received orphan drug designation from the FDA for its investigational medicine KRRO-110, aimed at treating Alpha-1 Antitrypsin Deficiency (AATD) [1][2] - KRRO-110 is the first RNA editing candidate from Korro's OPERA platform and is currently in Phase 1/2a clinical trials [2][4] - AATD is a genetic disorder that can lead to serious health issues, including pulmonary emphysema and hepatic cirrhosis [5] Company Overview - Korro Bio is a clinical-stage biopharmaceutical company focused on developing genetic medicines through RNA editing for both rare and prevalent diseases [6] - The company aims to leverage its proprietary RNA editing platform to create precise and transient genetic modifications, enhancing the specificity and tolerability of treatments [6] Clinical Development - The REWRITE study is a two-part clinical trial evaluating the safety and tolerability of KRRO-110 in up to 64 participants, with interim data expected in the second half of 2025 [4] - The study includes healthy adults and clinically stable AATD patients, focusing on pharmacokinetic and pharmacodynamic parameters to guide future dosing [4] Regulatory Insights - The FDA's orphan drug designation provides various incentives, including tax credits for clinical testing and potential market exclusivity post-approval [3] - This designation highlights the urgent need for new treatments for AATD patients, emphasizing the potential impact of KRRO-110 [2][3]