On Januaryn 6, 2022 2seventy bio, Inc. (NASDAQ:TSVT) reported that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A (Press release, 2seventy bio, JAN 6, 2022, View Source [SID1234598341]). This agreement builds upon a successful existing multi-year research collaboration between the two companies. Under the terms of this agreement, Novo Nordisk will have the option to license 2seventy bio’s proprietary mRNA-based megaTAL technology for the development of a new treatment approach for hemophilia A patients with the goal of offering a sustained therapeutic effect.

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"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 Philip Gregory, D. Phil., chief scientific officer, 2seventy bio. "We are also excited to announce a partnership between 2seventy bio and Genevant Sciences for the use of Genevant’s lipid nanoparticle (LNP) platform in our collaboration with Novo Nordisk."

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 Karin Conde-Knape, senior vice president, Global Drug Discovery, Novo Nordisk. "This partnership reflects Novo Nordisk’s commitment to utilizing novel technology platforms to advance truly disease-modifying therapies for people with serious chronic diseases."

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 $5 million and is eligible for near-term milestone and option exercise payments of up to $35 million, in addition to development, regulatory, and commercial milestones, as well as a royalty on net sales. Novo Nordisk will be responsible for funding all research and development activities.

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 Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation. "Our scientists have been at the forefront of the LNP field for more than 20 years, and we are excited for our innovative technology to be used for this important application with great unmet need."

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 $10 million, as well as development and commercialization milestones, and royalties in the mid-single digits on future product sales.

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.