Metagenomi advances gene editing therapies into 2025

Published 2025-01-16, 09:02 a/m
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EMERYVILLE, Calif. - Metagenomi, Inc. (NASDAQ: MGX), a biotechnology firm specializing in precision genetic medicines trading at $2.80 per share, has reported significant progress in its therapeutic development programs, with multiple milestones achieved in 2024 and key goals set for 2025 and 2026. According to InvestingPro data, the company maintains a strong balance sheet with more cash than debt, though it's currently experiencing rapid cash burn as it advances its development programs. The company's advancements include the development of MGX-001, a potential treatment for hemophilia A, and strides in cardiometabolic disease programs in collaboration with Ionis Pharmaceuticals (NASDAQ:IONS).

In the past year, Metagenomi declared MGX-001 as a wholly-owned development candidate and initiated discussions with the FDA while commencing Good Manufacturing Practice (GMP) activities. At the American Society of Hematology's annual meeting in December 2024, the company presented data demonstrating sustained Factor VIII activity in a nonhuman primate study, which has continued for over 16 months. The company's financial health score from InvestingPro stands at "FAIR," with a notably strong current ratio of 6.18, indicating robust short-term liquidity to support its development programs.

Looking ahead to 2025, Metagenomi aims to finalize its nonhuman primate durability study and continue efforts to secure an Investigational New Drug (IND) status. The company also plans to engage in pre-IND and ex-US regulatory meetings to support IND and Clinical Trial Application submissions for MGX-001 in 2026.

In the realm of secreted protein deficiencies, Metagenomi has identified targets and achieved in vivo proof-of-concept in rodents using an approach similar to that in MGX-001. The company anticipates demonstrating nonhuman primate proof-of-concept for a lead target in 2025, with the goal of nominating a development candidate in 2026.

Cardiometabolic programs, part of a collaboration with Ionis Pharmaceuticals, have progressed with all four Wave 1 programs reaching lead optimization and demonstrating in vivo proof-of-concept in rodents. These programs address conditions such as transthyretin amyloidosis and refractory hypertension. In 2025, Metagenomi expects to nominate one or two development candidates from these programs and reveal the therapeutic indications for the remaining development programs.

Technological advancements by Metagenomi in 2024 include the presentation of compact SMART nucleases and novel Adenine Base Editors, with the potential to target over 95% of the human genome. The company plans to continue developing its early-stage pipeline with multiple IND filings anticipated.

Metagenomi has also announced the appointment of Eric Bjerkholt, CFO of Mirum Pharmaceuticals (NASDAQ:MIRM), Inc., to its Board of Directors, where he will serve on the Audit and Compensation committees.

This news is based on a press release statement and includes forward-looking statements subject to risks and uncertainties that could affect the company's business and stock value. These statements are not guarantees of future performance and involve known and unknown risks. Analysts maintain an optimistic outlook, with price targets ranging from $14 to $25, significantly above current trading levels. InvestingPro subscribers have access to 13 additional investment tips and comprehensive financial analysis tools to better evaluate the company's potential. The stock currently appears undervalued according to InvestingPro's Fair Value analysis.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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