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Metagenomi, Inc. (Nasdaq: MGX) is a leading precision genetic medicines company dedicated to developing curative therapeutics for patients through its advanced, metagenomics-derived genome editing toolbox. The company's innovative platform includes programmable nucleases, base editors, and both RNA and DNA-mediated integration systems such as prime editing systems and CRISPR-associated transposases (CASTs). Metagenomi's mission is to address the diverse mutations causing genetic diseases, which have largely been unapproachable by previous genome engineering methods.
In 2023, Metagenomi achieved significant milestones, such as demonstrating preclinical proof of concept in nonhuman primates for its lead Hemophilia A program. This included the successful integration of a functional Factor VIII gene, resulting in clinically relevant Factor VIII levels over a 4.5-month follow-up period. The company is on track to nominate a Development Candidate for this program by mid-2024 and aims to present 12-month durability data later in the year.
Key partnerships have also advanced, with Ionis Pharmaceuticals completing target selection for multiple collaboration efforts, focusing on diseases like Transthyretin Amyloidosis and Cardiovascular Disease. Metagenomi's technology platform continues to make groundbreaking advancements, particularly with its Big RIGS (RNA-mediated integration systems) and CAST systems, highlighting the company's capacity to perform large, targeted gene integrations.
The company's proprietary SMART base editors, some of the smallest in the industry, underline its leadership in genome editing. These editors have been engineered to be highly efficient and are suitable for a broad range of therapeutic applications.
In early 2024, Metagenomi announced the successful closing of its initial public offering, raising approximately $93.75 million. This funding will support the company’s continued research and development efforts, including advancing two INDs and nominating two additional Development Candidates. Metagenomi's strong financial position, with $271.2 million in cash and marketable securities as of December 31, 2023, plus the IPO proceeds, ensures a robust cash runway to support its ambitious goals.
Corporate infrastructure has been bolstered with key executive appointments, including Sarah Noonberg as Chief Medical Officer, Luis Borges as Chief Scientific Officer, and Pamela Wapnick as Chief Financial Officer. These strategic hires are pivotal in driving the company's clinical development and operational expansion.
Metagenomi recently regained full control over its base editing and RIGS technologies, previously under collaboration with Moderna, allowing the company to further its mission in developing gene editing therapies for conditions with significant unmet needs like Alpha-1 Antitrypsin Deficiency and Wilson’s Disease.
For more information, please visit Metagenomi's official website.
Metagenomi (Nasdaq: MGX) has announced promising 12-month durability data for its lead hemophilia A program, MGX-001. The preclinical study in nonhuman primates (NHPs) demonstrated durable Factor VIII (FVIII) activity levels through twelve months, with two NHPs showing normal/near-normal levels (82% and 41%) and one in the mild hemophilia range (9%). The treatment, involving a single dose of AAV followed by an LNP containing Metagenomi's novel nuclease, was generally well-tolerated.
Key findings include:
- Consistent FVIII activity levels from 3-6 months to 9-12 months
- Gene integration in liver biopsies correlating with FVIII activity
- Program on track for IND filing in 2026
Metagenomi aims to provide a one-time, curative treatment for adults and children with hemophilia A and plans to leverage this platform for other secreted protein disorders.
Metagenomi (Nasdaq: MGX) presented advancements in its AI-enabled SMART Editing Platform at the CRISPR Frontiers Conference. The company showcased AI-generated compact SMART nucleases with robust genome editing activity in mammalian cells. These tools, fitting within size-constrained delivery technologies, may enable broader disease targeting.
Key highlights include:
- AI-based gene editing variants showing high efficiency in mammalian cells
- Compact base editors less than 1,000 amino acids long
- De novo, AI-generated synthetic nucleases demonstrating robust activity
- Rational engineering achieving saturating levels of editing in human cells
Metagenomi's approach combines its metagenomics database of over 11 billion proteins with AI to create novel gene editing tools, potentially accelerating drug development and opening new therapeutic applications.
Metagenomi (Nasdaq: MGX), a precision genetic medicines company, has announced its participation in two upcoming investor conferences. The company will present at the Wells Fargo Healthcare Conference on September 4, 2024, and the H.C. Wainwright 26th Annual Global Investment Conference on September 9, 2024.
Key executives, including CEO Brian Thomas, CMO Dr. Sarah Noonberg, SVP Preclinical Alan Brooks, and CFO Pamela Wapnick, will represent Metagenomi at these events. The presentations will be available via live webcast on the company's investor relations website, with replays accessible for a time.
Metagenomi specializes in developing curative therapeutics using its proprietary, metagenomics-derived toolbox. The company's comprehensive genome editing toolkit includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems, positioning Metagenomi to address a wide range of genetic mutations.
Metagenomi (Nasdaq: MGX) reported Q2 2024 financial results and business updates. Key highlights include:
1. Declared MGX-001 as lead candidate for hemophilia A treatment
2. All Wave 1 Ionis collaboration programs advancing in lead optimization
3. Achieved milestone with Affini-T related to licensed technology
4. $299.9M in cash and equivalents; runway into 2027
5. R&D expenses increased to $28.3M from $22.7M YoY
6. G&A expenses rose to $8.6M from $6.6M YoY
The company plans to present 12-month NHP durability study data for MGX-001 in September 2024, accelerated from late 2024. Metagenomi aims to file an IND for hemophilia A in 2026 and nominate 1-2 development candidates for cardiometabolic programs in 2025.
Metagenomi (Nasdaq: MGX), a precision genetic medicines company, will present at the 2024 Jefferies Global Healthcare Conference on June 5, 2024. CEO Brian C. Thomas will deliver the presentation at 11:30 a.m. Eastern Time, and the company will also host one-on-one investor meetings during the conference from June 5-6. The presentation will be live-streamed on Metagenomi's website. A replay will be available for 30 days post-event.
Metagenomi specializes in developing curative therapeutics using a metagenomics-derived gene editing toolbox. Their technology harnesses microbial evolution over four billion years to create tools capable of correcting genetic mutations in the genome. The toolbox includes programmable nucleases, base editors, and RNA and DNA integration systems.
Metagenomi (MGX) announced its Q1 2024 financial results, revealing a cash position of $327.4 million, sufficient to support operations until 2027. The company's key scientific presentations showcased advancements in gene editing for Hemophilia A and other genetic conditions. At the World Federation of Hemophilia Congress, Metagenomi highlighted Factor VIII expression within the therapeutic range in non-human primates, with a development candidate expected by mid-2024. At the ASGCT meeting, they presented data demonstrating a five-fold increase in genome targetability using MGX base editing and successful large gene integration in human cells. Additionally, Metagenomi regained full rights to its gene editing technologies, previously held by ModernaTX. Financially, the company reported increased R&D expenses ($31.4M) and G&A expenses ($8.8M), leading to a net loss of $25.1 million, compared to $16.1 million in Q1 2023.
Metagenomi, Inc. (Nasdaq: MGX) has regained full control of its gene editing technologies, including base editors and RIGS systems, previously under exclusive rights with Moderna, Inc. The termination of the collaboration on primary hyperoxaluria type 1 (PH1) has allowed Metagenomi to focus on developing therapies for Alpha-1 antitrypsin deficiency and Wilson’s disease, enhancing its strategic flexibility and control. The company aims to accelerate the advancement of its gene editing technologies and engage in target-specific applications through partnerships.
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