Metagenomi

Metagenomi is excited to announce preclinical data for our lead program in hemophilia A, demonstrating durable factor VIII (FVIII) activity levels through twelve months of follow up. Hemophilia A is the most common X-linked inherited bleeding disorder, caused by a large variety of mutations in the FVIII gene leading to a loss of functional FVIII protein. Our goal is to provide a one-time, curative treatment for adults and children with hemophilia A. For further information, be sure to read the full press release linked below. Additionally, Metagenomi management will be holding a conference call tomorrow, September 4th at 8:30am EST. For login information, please visit Metagenomi's investor page on the Company website. Press Release: https://lnkd.in/g3XAnmgQ

We are excited to announce Metagenomi's poster presentation at the Cold Spring Harbor Laboratory CRISPR Frontiers Conference, highlighting our AI-enabled SMART editing platform. While there is no shortage of naturally occurring proteins that we can discover using our metagenomics platform, AI is one of several approaches we leverage to generate novel systems capable of highly efficient in vivo gene editing. Read our latest press release here for more: https://lnkd.in/gPbVgGTE

Metagenomi is looking forward to presenting at the upcoming Wells Fargo and HC Wainwright investor conferences. For further details, please refer to the press release here: https://lnkd.in/gum7K-pv

Today we announced business updates along with our Q2 financial results. You can find the key highlights below, with the full details available in the press release here: https://lnkd.in/gPq3VE6t

Today we announced Metagenomi's data presentations at recent scientific conferences and first quarter 2024 financial results. See the press release here: https://lnkd.in/gwAkW9z3

Metagenomi is looking forward to sharing scientific updates at the American Society of Gene and Cell Therapy’s 27th Annual Meeting in Baltimore, MD. Be sure to stop by our poster sessions to connect with our scientists and learn more.

Tomorrow, Metagenomi’s Head of Preclinical, Alan Brooks, Ph.D., will give an oral presentation at the World Federation of Hemophilia (WFH) World Congress. If you are attending in-person, be sure to stop by and hear Dr. Brooks speak on Metagenomi’s novel, durable, knock-in approach for expression of Factor VIII.

Today is World Hemophilia Day. There are estimated to be nearly 30,000 patients with hemophilia A in the United States and more than 500,000 patients with hemophilia A globally. Hemophilia A is the most common X-linked inherited bleeding disorder and is caused by mutations in the Factor VIII (“FVIII”) gene leading to loss of functional FVIII protein that impacts the body’s ability to form normal clots in response to injury. At Metagenomi, we are advancing our lead program in Hemophilia A— with a novel, durable , knock-in approach for expression of Factor VIII. Our goal is to harness the power of our proprietary metagenomics platform to create curative genetic medicines for patients. The progress we have made on this program not only validates the efficiency and specificity of our novel nucleases in rodent and NHP models, but also supports our ongoing efforts with other large gene integration approaches. Today and every day, we hope to spread awareness for patients everywhere living with Hemophilia A.

Our co-authored publication with the Taylor Lab at UT Austin, “DNA targeting by compact Cas9d and its resurrected ancestor,” is live on bioRxiv. Read on to learn more about the structure and biochemistry of the novel SMART nuclease MG34-1, also known as Cas9d. Findings in this latest publication show SMART protein activity in mammalian cells - These results overcome a significant hurdle for translation of the Cas9d family and demonstrates the incredible potential of the Cas9d family for AAV delivery for human genome editing applications. Finally, we identify the first Cas9d smaller than 800 amino acids exhibiting robust nuclease activity in mammalian cells. https://lnkd.in/gxQSmH-X

Today, Metagenomi announced that we will be presenting at upcoming conferences. Read the press release here: https://lnkd.in/g8zr4WVi