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]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____________________ to _____________________ Commission File Number: 001-39062 Korro Bio, Inc. (Exact name of registrant as specified in its charter) Delawa ...

Exhibit 99.1 Korro Reports First Quarter 2025 Financial Results and Provides Business Updates CAMBRIDGE, Mass., May 7, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical- stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, today reported financial results for the first quarter of 2025 and provided a business update. Ram Aiyar, Ph.D., CEO and President of Korro, said, "We remain on tr ...

—Interim readout from Phase 1/2a REWRITE clinical trial of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD) on track for the second half of 2025 —Streamlining operations and focusing on delivering potential value generating program milestones, with workforce reduction of approximately 20% and cost reductions expected to extend cash runway into 2027 —Second development candidate expected to be announced in 2025 —Ended first quarter 2025 with $139.0 million in cash, cash equivalents and marketable secur ...

"Korro's unique RNA-editing platform has the potential to bring transformative genetic medicines to patients with rare and highly prevalent diseases and provide hope where there is significant unmet need," said Dr. Vincent. "I look forward to supporting the mission at Korro and joining its talented management team." CAMBRIDGE, Mass., April 01, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (NASDAQ:KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines base ...

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]

Core Insights - Korro Bio, Inc. is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines through RNA editing for both rare and prevalent diseases [3] Company Overview - Korro Bio utilizes a proprietary RNA editing platform to create a portfolio of differentiated programs aimed at harnessing the body's natural RNA editing process, allowing for precise and transient single base edits [3] - The company aims to enhance the precision and tunability of genetic medicines by editing RNA instead of DNA, which may lead to increased specificity and improved long-term tolerability [3] - Korro plans to leverage its proprietary platform along with established delivery modalities, manufacturing expertise, and regulatory pathways of approved oligonucleotide drugs to bring its medicines to patients [3] Upcoming Events - Ram Aiyar, Ph.D., CEO and President of Korro, will present at the TD Cowen 45th Annual Health Care Conference on March 4, 2025, at 11:50 a.m. ET [1] - Todd Chappell, COO, will participate in one-on-one investor meetings at the same conference [1]