2seventy Bio, Novo Nordisk Expand Partnership to Develop Gene Editing

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by Steve Bryson, PhD |

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2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia A.

The new venture adds to an ongoing partnership between 2seventy’s parent company, Bluebird Bio, and Novo Nordisk, which focused on finding new gene therapy candidates.

Under this agreement, Novo Nordisk has the option to exclusively license 2seventy Bio’s proprietary mRNA-based megaTAL technology — customizable enzymes designed to recognize and cut specific DNA regions.

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This gene-editing approach has the potential to correct the faulty F8 gene that causes hemophilia A and allow for the proper production of the blood-clotting protein factor VIII (FVIII).

“This collaboration aims to develop the first direct in vivo application [in the organism] of our megaTAL technology,” Philip Gregory, PhD, chief scientific officer at 2seventy Bio, said in a press release. “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.”

In return, 2seventy Bio will receive a $5 million upfront payment and is eligible for near-term milestone payments, as well as exercised stock option amounts of up to $35 million. These funds are in addition to development, regulatory, and commercial milestones, and royalties on sales.

“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 at Novo Nordisk, which will fund all research and development activities.

“This partnership reflects Novo Nordisk’s commitment to utilizing novel technology platforms to advance truly disease-modifying therapies for people with serious chronic diseases,” Conde-Knape added.

In support of the collaboration with Novo Nordisk, 2seventy has also partnered with Genevant Sciences to access Genevant’s lipid nanoparticle (LNP) system to efficiently deliver megaTAL mRNA to cells to the liver where FVIII is produced.

“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, PhD, president and CEO at Genevant Sciences. “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 this agreement, 2seventy Bio can license Genevant’s LNP technology for megaTAL mRNA to advance treatments for hemophilia A, while Genevant may receive upfront payments, as well as near-term option exercise payments totaling $10 million. In addition, Genevant is eligible for development and commercialization milestones and royalties from sales.

Recently, 2seventy split off from Bluebird Bio to focus mainly on developing immune-based cancer therapies using megaTAL technology.

“We view this work as further validation and a natural extension of our technology platform,” Gregory added. “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 [outside the organism] applications that can enhance our oncology [cancer] pipeline.”