Core Insights - Metagenomi, Inc. is set to present at two significant scientific meetings, showcasing its advancements in gene editing technologies [1][2] - The company utilizes an AI-driven metagenomics platform to develop curative therapeutics, aiming to correct genetic mutations across the genome [3] Presentation Details - At TIDES USA 2025, Metagenomi will deliver an oral presentation titled "AI-Guided Metagenomic Discovery of Compact CRISPR Systems for In Vivo Therapeutic Genome Editing" on May 22, 2025 [2] - The company will also present three posters at the ASGCT 2025 Annual Meeting, focusing on various aspects of in vivo genome editing and CRISPR systems on May 13, 2025 [2] Company Overview - Metagenomi is dedicated to developing precision genetic medicines, leveraging four billion years of microbial evolution to create novel genome editing tools [3] - The company's toolbox includes programmable nucleases, base editors, and advanced integration systems, positioning it to address a wide range of genetic mutations [3]

Exhibit 99.1 Metagenomi Reports Business Updates and Full Year 2024 Financial Results Hemophilia A presentation at American Society of Hematology (ASH) 66th Annual Meeting demonstrated sustained Factor VIII (FVIII) activity in nonhuman primate (NHP) study for more than 16 months Leveraged hemophilia A albumin platform to achieve in vivo proof-of-concept in multiple secreted protein deficiencies to support wholly-owned follow-on program Progressed four Wave 1 Ionis targets to lead optimization with plans to ...

Core Insights - Metagenomi, Inc. reported significant advancements in its hemophilia A program, demonstrating sustained Factor VIII (FVIII) activity in nonhuman primate studies for over 16 months, supporting the durability of its gene editing therapy [1][7] - The company is well-capitalized with $248.3 million in cash and equivalents as of December 31, 2024, providing a cash runway anticipated to support operations into 2027 [1][10] - Metagenomi plans to submit its first Investigational New Drug (IND) application for hemophilia A in 2026 and aims to nominate one to two development candidates from its Wave 1 Ionis collaboration programs in 2025 [2][14] Hemophilia A Program - The company has declared MGX-001 as a development candidate for hemophilia A, which includes a bioengineered FVIII construct with higher activity levels compared to the wild type [7] - An oral presentation at the American Society of Hematology (ASH) highlighted the sustained FVIII activity in nonhuman primate studies, reinforcing the potential of MGX-001 [7] - Plans are in place to release final FVIII durability and related preclinical study data in the first half of 2025 [14] Secreted Protein Deficiencies - Metagenomi has identified targets for wholly-owned therapeutic programs leveraging the gene integration approach used in MGX-001, achieving in vivo proof-of-concept in rodents across three secreted protein targets [4] - The company plans to demonstrate nonhuman primate proof-of-concept for its lead secreted protein deficiency target in 2025 and nominate a development candidate in 2026 [9] Cardiometabolic Indications - The company advanced four Wave 1 Ionis collaboration programs to lead optimization, including targets for transthyretin amyloidosis and refractory hypertension, achieving in vivo proof-of-concept in rodents [8] - Demonstrated a 95% protein knockdown in spontaneous hypertensive rats, showcasing the progress made in the collaboration [8] Financial Performance - For the full year ended December 31, 2024, Metagenomi reported research and development expenses of $109.2 million, an increase from $94.4 million in 2023 [10] - General and administrative expenses rose to $32.0 million in 2024 from $28.8 million in 2023 [11] - The net loss for 2024 was $78.1 million, compared to a net loss of $68.3 million in 2023, with a net loss per share of $2.36 [20]

Financial Performance - Collaboration revenue increased to $52.3 million for the year ended December 31, 2024, compared to $44.8 million in 2023, reflecting a growth of approximately 33.3%[558] - Collaboration revenue increased by $7.5 million, from $44.8 million in 2023 to $52.3 million in 2024, primarily driven by an $8.5 million increase from the Ionis Agreement[572] - The net loss for 2024 was $78.1 million, compared to a net loss of $68.3 million in 2023, reflecting an increase of $9.8 million[581] - Total operating expenses rose to $141.2 million in 2024, up $17.9 million from $123.2 million in 2023, with research and development expenses accounting for $109.2 million, an increase of $14.8 million[574] - Total other income, net, decreased by $12.9 million, from $18.3 million in 2023 to $5.3 million in 2024, primarily due to a loss of $9.2 million on long-term investments[576] - The provision for income taxes changed from an expense of $8.0 million in 2023 to a benefit of $5.5 million in 2024, reflecting a strategic tax credit carryback[577] - The company has an accumulated deficit of $223.0 million as of December 31, 2024, indicating ongoing financial challenges[581] Cash Flow and Liquidity - Cash used in operating activities was $109.1 million for 2024, consisting mainly of the net loss and changes in operating assets and liabilities[586] - As of December 31, 2024, the company had $248.3 million in cash, cash equivalents, and available-for-sale marketable securities, sufficient to fund projected operating expenses for at least the next 12 months[582] - Net cash used in operating activities for the year ended December 31, 2023 was $91.4 million, primarily due to a net loss of $68.3 million and a net reduction of $26.4 million in net operating assets and liabilities[587] - Net cash provided by investing activities for the year ended December 31, 2023 was $45.7 million, consisting of $55.5 million in net maturities of available-for-sale marketable securities, offset by $9.8 million of purchases of property and equipment[589] - Net cash provided by financing activities for the year ended December 31, 2024 was $84.0 million, attributed to net proceeds from the issuance of common stock in the IPO[590] - Net cash used in investing activities for the year ended December 31, 2024 was $88.2 million, primarily due to net purchases of available-for-sale securities of $84.7 million[588] - Net cash provided by financing activities for the year ended December 31, 2023 was $1.0 million, consisting of $4.3 million of net cash proceeds from the issuance of Series B-1 preferred redeemable convertible preferred stock[591] Research and Development - The investigational therapy for hemophilia A demonstrated durable FVIII activity levels in non-human primates over a 16.5-month period, with no significant safety findings reported[547] - The company plans to conduct pre-IND and ex-U.S. regulatory meetings in 2025 and aims to complete IND and clinical trial application filings for MGX-001 in 2026[547] - Research and development expenses are expected to increase substantially as the company continues to invest in its platform and advance clinical trials[562] General and Administrative Expenses - General and administrative expenses are anticipated to rise due to increased headcount and costs associated with being a public company[564] - General and administrative expenses increased to $32.0 million in 2024, up $3.2 million from $28.8 million in 2023, primarily due to increased stock-based compensation[575] Collaborations and Partnerships - The company has entered into collaborations with Ionis Pharmaceuticals and Affini-T Therapeutics to develop gene editing therapies for cardiometabolic diseases and cancer, respectively[548][555] - The company has not generated any revenue from product sales and does not expect to do so in the foreseeable future, relying instead on collaboration agreements for revenue[556] Corporate Actions - The company completed its initial public offering (IPO) on February 13, 2024, issuing 6,250,000 shares at $15.00 per share, resulting in net proceeds of approximately $80.7 million after costs[552] - The Reorganization and reverse stock split were completed on January 24, 2024, and January 26, 2024, respectively, with a 1-for-1.74692 ratio for the stock split[549][551] Tax and Deferred Assets - As of December 31, 2024, the company had net operating loss carryforwards of $45.6 million for federal and $118.3 million for state income tax purposes, available to reduce future taxable income[567] - The company assesses the need for a valuation allowance against deferred tax assets based on various factors, including projected future taxable income and historical earnings experience[609] - The company recognizes tax benefits related to uncertain tax positions when it is more likely than not that a tax position will be sustained during an audit[610]

Core Insights - Metagenomi has developed a novel, compact CAST system for large, targeted gene integration into the human genome, which is a significant advancement in the field of gene editing [1][2][4] - The CAST system is designed to address complex genetic diseases caused by loss of function mutations, such as Wilson's disease, cystic fibrosis, and Duchenne Muscular Dystrophy [2][4] - The technology utilizes an AI-driven metagenomics platform to facilitate the discovery and application of the CAST system in human cells [1][3] Company Developments - The compact CAST system allows for the integration of multiple gene cargoes at a known safe-harbor site in the human genome, showcasing its site-specific and programmable capabilities [3][4] - The system simplifies delivery through a single 'all-in-one' mRNA design, representing a first-in-class approach in the industry [1][4] - Future advancements in large gene integration technology are anticipated in 2025, with ongoing efforts to optimize the system for clinical applications [1][4] Industry Impact - The findings from Metagenomi's research highlight the potential of compact CAST systems to overcome limitations in large gene integration, paving the way for precision genetic medicines [4] - The technology is positioned to unlock the full potential of genome editing for patients, addressing a wide range of genetic mutations [5]

Core Viewpoint - Metagenomi, Inc. is actively participating in the TD Cowen's 45th Annual Health Care Conference, showcasing its commitment to developing curative therapeutics through its proprietary gene editing toolbox [1]. Company Overview - Metagenomi is a precision genetic medicines company focused on curative therapeutics using an AI-driven metagenomics platform [3]. - The company leverages four billion years of microbial evolution to discover novel editing tools capable of correcting any genetic mutation in the genome [3]. - Its genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems, such as prime editing systems and CAST [3]. - Metagenomi's proprietary toolbox is designed to access the entire genome and select optimal tools for effective genome editing [3]. Event Participation - Brian C. Thomas, PhD, CEO and founder of Metagenomi, will engage in a fireside chat at the conference on March 5, 2025, from 1:10-1:40 p.m. ET in Boston [1]. - The company will also host one-on-one investor meetings during the conference [1].

Core Insights - Metagenomi, Inc. is advancing its gene editing candidate MGX-001 for hemophilia A, with regulatory interactions anticipated in 2025 and a focus on achieving proof-of-concept for secreted protein deficiencies [1][2][6] - The company is on track to nominate one to two development candidates from its collaboration with Ionis in 2025, targeting large cardiometabolic indications [1][12] - Metagenomi's cash runway is expected to support its operational plans into 2027, indicating financial stability for ongoing projects [1] Hemophilia A Program - MGX-001 has achieved sustained Factor VIII (FVIII) activity in a nonhuman primate study over 16 months, demonstrating its potential efficacy [6] - The company has engaged with the FDA for initial regulatory discussions and has initiated GxP manufacturing activities for MGX-001 [6] - Plans for 2025 include finalizing the ongoing nonhuman primate durability study and continuing Investigational New Drug (IND) enabling efforts [6] Secreted Protein Deficiencies - In 2024, Metagenomi identified targets for wholly-owned therapeutic programs leveraging the albumin approach used in MGX-001 [4] - The company anticipates demonstrating proof-of-concept for its lead secreted protein deficiency target in nonhuman primates in 2025 [7] Cardiometabolic Programs - Metagenomi has advanced all four Wave 1 Ionis collaboration programs to lead optimization, achieving in vivo proof-of-concept in rodents [12] - The company plans to nominate development candidates from these programs in 2025 and disclose remaining therapeutic indications [12] Technology Development - Metagenomi has made advancements in its gene editing toolbox, including next-generation base editors and ultra-small nucleases [2][12] - The company presented novel Adenine Base Editors (ABEs) that target over 95% of the human genome, demonstrating high specificity and no adverse effects on cell health [12]

Core Insights - Metagenomi, Inc. announced the appointment of Eric Bjerkholt, CFO of Mirum Pharmaceuticals, to its Board of Directors, effective January 27, 2025 [1][2] - Bjerkholt's extensive experience in the pharmaceutical industry and business development is expected to enhance Metagenomi's efforts in developing curative genetic therapies [2] - Metagenomi utilizes a proprietary metagenomics-derived toolbox to create precision genetic medicines, aiming to correct genetic mutations across the genome [3] Company Overview - Metagenomi is focused on developing curative therapeutics using a comprehensive toolbox derived from metagenomics, which studies genetic material from the environment [3] - The company's toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems, positioning it to access the entire genome for optimal editing solutions [3] Leadership Experience - Eric Bjerkholt has a strong background in financial leadership, having served as CFO at multiple pharmaceutical companies, including Mirum Pharmaceuticals and Chinook Therapeutics [2] - His previous roles include overseeing financial reporting, planning, budgeting, and investor relations, contributing to his capability to support Metagenomi's strategic goals [2]

Core Viewpoint - Metagenomi, Inc. is set to present at the 43rd Annual J.P. Morgan Healthcare Conference, highlighting its commitment to developing curative therapeutics through its proprietary gene editing toolbox [1]. Company Overview - Metagenomi is a precision genetic medicines company focused on curative therapeutics using a comprehensive metagenomics-derived toolbox [3]. - The company utilizes metagenomics to explore four billion years of microbial evolution, aiming to discover novel editing tools for correcting genetic mutations across the genome [3]. - Its genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems, such as prime editing systems and CAST [3]. - Metagenomi believes its diverse and modular toolbox allows access to the entire genome, optimizing genome editing potential for patients [3]. Event Details - Brian C. Thomas, PhD, CEO and founder of Metagenomi, will present on January 15, 2025, at 4:30 p.m. PT in San Francisco [1]. - A live webcast of the presentation will be available on the company's investor website, with a replay accessible for a limited time [2].

Core Insights - Metagenomi, Inc. is focused on developing curative therapeutics using its proprietary gene editing toolbox, emphasizing the precision and safety of its technologies [1][2][3] Group 1: Gene Editing Technologies - MGX-001, a candidate for hemophilia A, utilizes the MG29-1 nuclease and shows no identifiable off-target editing, confirmed through various assays [2] - The MG29-1 nuclease targeting the albumin safe harbor locus demonstrated no evidence of translocations in primary human hepatocytes [2] - Metagenomi's adenine base editor (ABE) for ex vivo cell therapy showed no detectable translocations and no significant genomic base composition differences in primary T-cells compared to unedited cells [2] Group 2: Company Overview - Metagenomi leverages metagenomics to develop a comprehensive toolbox for genome editing, which includes programmable nucleases, base editors, and integration systems [3] - The company aims to correct any type of genetic mutation found in the genome, utilizing four billion years of microbial evolution [3] - Metagenomi's diverse and modular toolbox is designed to access the entire genome and select optimal tools for effective genome editing [3]