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]

Core Viewpoint - Rosen Law Firm is reminding investors who purchased Metagenomi, Inc. stock during its IPO of the upcoming lead plaintiff deadline for a class action lawsuit [1] Group 1: Class Action Details - Investors who bought Metagenomi stock between February 9 and 13, 2024, may be eligible for compensation without any out-of-pocket fees through a contingency fee arrangement [1] - A class action lawsuit has already been filed, and interested parties must move the Court to serve as lead plaintiff by November 25, 2024 [2] - The Rosen Law Firm emphasizes the importance of selecting qualified legal counsel with a successful track record in securities class actions [3] Group 2: Company Background and Allegations - Metagenomi, described as a "genetic medicines company," had a collaboration with Moderna, which was highlighted during its IPO [4] - The company completed its IPO on February 13, 2024, selling 6.25 million shares at $15 per share [4] - The collaboration with Moderna was terminated on May 1, 2024, leading to claims that investors suffered damages when the true details were revealed [4]

Core Viewpoint - Rosen Law Firm is reminding investors who purchased Metagenomi, Inc. stock during its IPO of the upcoming lead plaintiff deadline for a class action lawsuit [1] Group 1: Class Action Details - Investors who bought Metagenomi stock during the IPO from February 9 to 13, 2024, may be eligible for compensation without any out-of-pocket costs through a contingency fee arrangement [1] - A class action lawsuit has already been filed, and interested parties must move the Court to serve as lead plaintiff by November 25, 2024 [2][5] - The lawsuit claims that investors suffered damages after Metagenomi announced the termination of its collaboration with Moderna, which was a key aspect of its IPO presentation [4] Group 2: Company Background - Metagenomi is described as a "genetic medicines company" that had a significant collaboration with Moderna, including a Strategic Collaboration and License Agreement established on October 29, 2021 [4] - The company completed its IPO on February 13, 2024, selling 6.25 million shares at a price of $15 per share [4]

Core Viewpoint - A securities class action lawsuit has been filed against Metagenomi Inc. for allegedly misleading investors regarding its business prospects and relationship with Moderna during the specified Class Period from February 6, 2024, to September 26, 2024 [1]. Company Overview - Metagenomi is a genetic medicines company based in Emeryville, CA, with a significant collaboration with Moderna, a leading Covid-19 vaccine company [3]. - The collaboration included a Strategic Collaboration and License Agreement established on October 29, 2021, which involved multiple four-year research programs [3]. IPO and Stock Performance - Metagenomi completed its initial public offering (IPO) on February 13, 2024, selling 6.25 million shares at $15 per share [4]. - Following the announcement of the termination of the collaboration with Moderna on May 1, 2024, Metagenomi's shares dropped from $7.04 to $6.17 within a day and continued to decline, trading slightly above $2.00 at the time of the lawsuit filing [5].