2seventy bio Announces Expanded Collaboration Agreement With Novo Nordisk to Continue Development of in vivo Gene Editing Approach
Collaboration further validates 2seventy bio’s megaTAL™ gene editing platform with potential applicability across company’s oncology pipeline
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2seventy bio and Genevant Sciences announce collaboration for lipid nanoparticle delivery of megaTAL mRNA
“This collaboration aims to develop the first direct in vivo application of our megaTAL technology. We are excited to explore this technology with Novo Nordisk, with the goal of developing a new potential therapeutic approach for patients with hemophilia A. Moreover, we view this work as further validation and a natural extension of our technology platform. Based on what we continue to learn, this technology may play a potential role in expanding our gene editing platform toward future in vivo and ex vivo applications that can enhance our oncology pipeline,” said
The collaboration agreement with Novo Nordisk builds upon the original research collaboration signed between bluebird bio and Novo Nordisk in 2019, focused on identifying a development gene therapy candidate for people with hemophilia A. The collaboration utilizes 2seventy bio’s megaTAL technology that has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components. Hemophilia A is a genetic bleeding disorder resulting from defective Factor VIII.
“We are excited to continue our partnership with 2seventy bio to jointly develop a next-generation in vivo genome editing treatment, with the ultimate ambition of offering people with hemophilia A lifetime free of factor replacement therapy,” said
Under the terms of the agreement, Novo Nordisk will obtain the option to exclusively license 2seventy bio’s in vivo mRNA platform and gene editing technology for use in the treatment of patients with hemophilia A. 2seventy bio will receive an upfront payment of
Related to this collaboration, 2seventy bio has also entered into an agreement with Genevant Sciences for access to Genevant’s industry-leading LNP technology platform for use in 2seventy bio’s collaboration with Novo Nordisk for the treatment of patients with hemophilia A. 2seventy bio plans to use the Genevant LNP platform for efficient delivery of megaTAL mRNA to hepatocyte cells within the liver.
“We are very pleased that 2seventy bio has entrusted Genevant and our LNP platform with delivery for its important gene editing program in hemophilia A,” said
Under the terms of the agreement between 2seventy bio and Genevant, 2seventy bio obtained rights to license Genevant’s LNP technology for use with megaTAL mRNA products in the treatment of patients with hemophilia A. Genevant is eligible for upfront and near-term option exercise payments totaling
About megaTALs
MegaTALs are single-chain enzymes that combine the natural DNA recognition and cleavage processes of Homing Endonucleases (HEs) with the modular DNA binding properties of transcription activator-like (TAL) effectors. This protein fusion architecture allows the generation of highly specific and active nucleases in a compact format compatible with all current viral and non-viral cell delivery methods.
About Novo Nordisk A/S
Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in
About Genevant Sciences
Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industry’s most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Company’s scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Company’s LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. For more information, please visit www.genevant.com.
About 2seventy bio
Our name, 2seventy bio, reflects why we do what we do - TIME. Cancer rips time away and our goal is to work at the maximum speed of translating human thought into action – 270 miles per hour — to give the people we serve more time. We are building the leading immuno-oncology cell therapy company, focused on discovering new therapies and leveraging platform technologies that truly disrupt the cancer treatment landscape.
With a deep understanding of the human body’s immune response to tumor cells and how to translate cell therapies into practice, we’re applying this knowledge to deliver next generation cellular therapies that focus on a broad range of hematologic malignancies, including the first FDA-approved CAR T cell therapy for multiple myeloma, as well as solid tumors. Our research and development is focused on delivering therapies that are designed with the goal to “think” smarter and faster than the disease. Importantly, we remain focused on accomplishing these goals by staying genuine and authentic to our “why” and keeping our people and culture top of mind every day.
For more information, visit www.2seventybio.com.
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2seventy bio Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements”. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, including statements regarding: plans for the expansion of our oncology pipeline and the development, regulatory approval, manufacture or sale of our current or future product candidates; expectations regarding use of our megaTAL technology for the treatment of patients with hemophilia A and other genetic conditions; expectations regarding use of Genevant’s LNP technology for the treatment of patients with hemophilia A and other genetic conditions; and expectations regarding the benefits we may receive from our agreements with Novo Nordisk and Genevant. Such forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual results to differ materially from those express or implied by such statements, including: the risk that our megaTAL technology is not effective in addressing hemophilia A and other genetic diseases; the risk that Genevant’s LNP technology will not be able to provide safe and efficient delivery of megaTAL mRNA to hepatocyte cells within the liver; the risk that we will not realize anticipated benefits from our agreements with Novo Nordisk and Genevant; and other internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. We caution investors not to place considerable reliance on forward-looking statements contained in this press release. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in the Quarterly Report filed by 2seventy bio with the
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