Novo Nordisk buys hemophilia A program from 2seventy Bio

Deal spins off years-long research collaboration

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer.

2seventy could receive payments of up to $40 million in exchange for transferring the hemophilia A program and licensing the megaTAL technology, which focuses on tailored enzymes designed to recognize and cut specific DNA regions.

“We are pleased to announce the completion of this [asset purchase agreement] with Novo Nordisk as we believe it will provide the appropriate resources for both the team and the science behind this important program,” Chip Baird, 2seventy’s CEO, said in a company press release.

The agreement spins off a years-long collaboration that began with Novo Nordisk and Bluebird Bio, 2seventy’s former parent company. The partnership focused on hemophilia A and was expanded in 2022 to advance the gene-editing technology.

“Novo Nordisk has been a valued partner over the past five years, and we are confident that under their leadership, the promise of developing a new treatment approach for patients living with hemophilia A will continue to progress,” Baird said.

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“We have been working closely with 2seventy bio colleagues aligned with our collaboration and I am thrilled to welcome them to Novo Nordisk where we together will leverage the megaTAL technology for pioneering therapeutic applications,” said Karina Thorn, PhD, Novo Nordisk’s corporate vice president of global nucleic acid therapies research.

Hemophilia A is caused by mutations in the F8 gene, which provides instructions for making factor VIII (FVIII), a protein needed for the blood to clot. When FVIII is faulty or missing, the blood cannot clot properly. This means that patients experience prolonged bleeding episodes that may require lifelong replacement therapy.

Gene therapy is a one-time treatment that aims to deliver a healthy version of the F8 gene, usually aboard a viral vehicle, to provide the body with the correct instructions to make FVIII on its own. Another approach aims, instead, to edit the mutated F8 gene that causes hemophilia A.

The megaTAL technology is a gene-editing tool that combines the actions of compact, custom-made enzymes to cut DNA at specific regions, which is expected to make the editing very precise. It can be used with both viral and nonviral delivery vehicles.

“I am excited that we are expanding our genome editing technology platforms at Novo Nordisk,” Thorn said. “We are devoted to developing therapies with a curative outlook, including our continued development of a next-generation in vivo genome editing program aiming to offer individuals living with hemophilia A a lifetime free of factor replacement therapy.”

The agreement will allow 2seventy to focus exclusively on developing and marketing Abecma (idecabtagene vicleucel), a cell-based therapy approved to treat multiple myeloma that the company developed in collaboration with Bristol Myers Squibb.