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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 (MGX) reported Q3 2024 financial results and business updates, highlighting the nomination of MGX-001, their first development candidate for hemophilia A treatment. The company demonstrated successful proof-of-concept with durable Factor VIII activity levels over 12 months in nonhuman primates. Financial highlights include $274.6 million in cash and equivalents, with runway into 2027. R&D expenses were $26.3 million and G&A expenses were $7.6 million. The company expects one to two development candidate nominations in 2025 from their Ionis collaboration programs focusing on cardiometabolic indications.
Metagenomi (Nasdaq: MGX) announced that its abstract on Factor VIII gene insertion has been accepted for oral presentation at the American Society of Hematology (ASH) 66th Annual Meeting in December 2024. The presentation will showcase preclinical proof-of-concept data for their lead candidate MGX-001, which aims to provide a single, curative treatment for hemophilia A.
The research demonstrates durable Factor VIII expression in nonhuman primates, addressing current therapy limitations that fail to prevent breakthrough bleeding. The presentation, led by VP Preclinical Alan Brooks, PhD, is scheduled for December 9, 2024, at 5:30 PM in San Diego Convention Center.
Metagenomi presented groundbreaking results for its Adenine Base Editors (ABEs) at the European Society of Gene and Cell Therapy Annual Congress in Rome. The company's ABE platform demonstrated exceptional performance across key metrics: over 95% genome targetability, achieving 95% protein knockdown in primary T-cells through triplex editing, and highly specific on-target deamination with no detectable translocations.
The platform showed excellent tolerability with no adverse effects on cell viability or expansion. Notable advantages include compatibility with various non-viral methods of site-specific template integration, enabling an all-in-one editing solution for next-generation cell therapies. This positions Metagenomi's ABE technology as a promising tool for both in vivo genome editing programs and ex vivo cell therapy applications.
Metagenomi (Nasdaq: MGX) presented a poster at the AIChE 7th International Conference on CRISPR Technologies, highlighting their SMART adenine base editors (ABEs). These compact editors are small enough to be packaged into a single adeno-associated virus (AAV), potentially addressing a key challenge in in vivo gene editing.
The company's SMART platform demonstrates improved editing efficiency across multiple targets and cell types, enabling pursuit of neuromuscular targets. This approach could be advantageous for treating diseases like Duchenne muscular dystrophy, ALS, and Charcot-Marie-Tooth disease type 1A.
Metagenomi has optimized the SMART platform through structure-guided engineering and advanced artificial intelligence (AI) tools, showcasing the importance of leveraging multiple techniques to advance next-generation gene editing systems.
Metagenomi (Nasdaq: MGX), a precision genetic medicines company, has announced its participation in Chardan's 8th Annual Genetic Medicines Conference on September 30, 2024. The company will be involved in two key events:
1. A fireside chat from 2:30 PM to 3:00 PM ET, featuring CEO and Founder Brian Thomas, CMO Dr. Sarah Noonberg, and SVP Preclinical Alan Brooks.
2. A panel discussion titled "Genome editing for gene insertion and gene correction - where we are and what's next" from 5:00 PM to 5:30 PM ET, with CEO Brian C. Thomas participating.
A live webcast of the fireside chat will be available on the company's investor relations website, with replays of both events accessible for a time.
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.
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