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]

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]

Core Insights - Korro Bio, Inc. has initiated dosing in its REWRITE study for KRRO-110, targeting Alpha-1 Antitrypsin Deficiency (AATD) [1][2] - The REWRITE study aims to evaluate the safety and tolerability of KRRO-110 in up to 64 participants, with interim data expected in the second half of 2025 [2][7] - KRRO-110 is designed to restore therapeutic M-AAT protein levels by utilizing the body's endogenous ADAR enzyme, with a focus on both lung and liver manifestations of AATD [2][5] Company Overview - Korro is a clinical-stage biopharmaceutical company focused on developing genetic medicines using its proprietary RNA editing platform, OPERA™ [6][8] - The company is transitioning from a research organization to a clinical-stage drug development entity, with plans to advance three product candidates into the clinic by 2027 [4][6] - Korro's approach aims to enhance the precision and tunability of genetic medicines by editing RNA instead of DNA, potentially improving long-term tolerability [6][8] Product Development - KRRO-110 is the first product candidate from Korro's RNA editing platform, targeting the genetic disorder AATD caused by a mutation in the SERPINA1 gene [5][7] - The REWRITE study consists of two parts: single and multiple dose-escalating studies, with secondary endpoints focusing on pharmacokinetic and pharmacodynamic parameters [2][4] - The company is also advancing additional pipeline programs, including a partnership with Novo Nordisk in a cardiometabolic indication [3][4]

Company Overview - Korro Bio, Inc. is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on RNA editing for both rare and highly prevalent diseases [3] - The company utilizes a proprietary RNA editing platform to create a portfolio of differentiated programs that enable precise yet transient single base edits [3] - By editing RNA instead of DNA, Korro aims to enhance the precision and tunability of genetic medicines, potentially leading to increased specificity and improved long-term tolerability [3] Upcoming Events - Ram Aiyar, Ph.D., CEO and President of Korro, will present at the 43rd Annual J.P. Morgan Healthcare Conference on January 13, 2025, at 3:45 p.m. PT [1] - Vineet Agarwal, CFO, and Kemi Olugemo, M.D., FAAN, Chief Medical Officer, will also participate in the conference [1] - A live webcast of the presentation will be available on Korro's website, with a replay accessible for 30 days following the event [2]

Company Overview - Korro Bio, Inc. is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on RNA editing for both rare and prevalent diseases [4] - The company utilizes a proprietary RNA editing platform to create a portfolio of differentiated programs aimed at precise and transient single base edits [4] - By editing RNA instead of DNA, Korro aims to enhance the precision and tunability of genetic medicines, potentially leading to improved specificity and long-term tolerability [4] Upcoming Events - Management will participate in several investor conferences, including: - Piper Sandler 36th Annual Healthcare Conference on December 3, 2024, featuring a fireside chat with CEO Ram Aiyar [1] - 7th Annual Evercore ISI HealthCONx Conference on December 4, 2024, with another fireside chat by Dr. Aiyar [2] - Citi's 2024 Global Healthcare Conference on December 5, 2024, with 1x1 investor meetings [2] - Oppenheimer Movers in Rare Disease Summit on December 12, 2024, where Dr. Aiyar will participate in a panel discussion [3] Access to Presentations - Live webcasts of the fireside chats at the Piper Sandler and Evercore conferences will be available on Korro's website, with replays accessible for 30 days post-event [3]

Core Insights - Korro Bio, Inc. has received approval to initiate a Phase 1/2a clinical study of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD) in Australia [1][2] - The study aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of KRRO-110, with the first participant dosing expected in Q1 2025 and completion anticipated in 2026 [1][4] Company Overview - Korro Bio is focused on developing a new class of genetic medicines using its proprietary RNA editing platform, OPERA™, targeting both rare and prevalent diseases [6][7] - KRRO-110 is the first RNA editing oligonucleotide candidate from Korro, designed to edit the SERPINA1 RNA to restore normal AAT protein secretion [6] Study Details - The REWRITE study will involve up to 64 participants, including healthy adults and clinically stable AATD patients with the PiZZ genotype [3] - Secondary and exploratory endpoints will include pharmacokinetic and pharmacodynamic parameters to guide optimal dose selection for future studies [3] Clinical Expectations - Interim readout for the study is expected in the second half of 2025, focusing on participants with the PiZZ genotype [4] - The study's design aims to demonstrate the potential of KRRO-110 in patients at risk for serious lung and liver complications despite standard treatment [2][9]