2seventy bio, Inc. announced 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. 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. 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. 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 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. 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.