Early milestone in work for hem A gene-editing tool met: 2seventy bio

Preclinical 'proof of concept' work could to be worth $15M to company

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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2seventy bio announced that it has achieved a key preclinical milestone under its joint program with Novo Nordisk to develop a gene-editing tool to treat hemophilia A.

Details of the “proof of concept” research that met this milestone — reported to be worth $15 million to the company — were not disclosed. Preclinical study findings will be presented at a virtual Research and Development deep dive on May 19.

“We are excited about the continued progress in this program based on our internally developed in vivo gene editing platform,” Philip Gregory, PhD, chief scientific officer at 2seventy bio, said in a company press release. In vivo research is conducted inside a living organism, rather than in a lab dish.

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Creating ‘permanent FVIII production’ seen as ‘best approach for treatment’

Hemophilia is caused by the lack of certain blood clotting factors — proteins that help the blood to form clots to control bleeding — usually due to genetic mutations. In the case of hemophilia A, mutations in the F8 gene result either in the production of a faulty version of a blood clotting protein called factor VIII (FVIII) or cause its levels to drop. The altered or missing protein cannot clot blood as it should, ultimately leading to excessive bleeding.

2seventy bio and Novo Nordisk partnered to develop gene therapy candidates for hemophilia A in 2022, renewing a working relationship previously established between 2seventy bio’s parent company, bluebird bio, and Novo Nordisk.

The gene-editing approach aims to correct the faulty F8 gene that causes hemophilia A and restore production of a functional FVIII protein.

“Hemophilia A is a lifelong condition that is detected early; the best solution for the condition is to create permanent FVIII production for the lifetime of patients which is why we believe integrating the corrective transgene into the genome is the best approach for treatment,” Gregory said.

Under the agreement, 2seventy bio’s proprietary mRNA-based megaTAL technology and in vivo mRNA platform are to be used to develop gene-editing tools that could allow scientists to precisely silence, edit, or insert genetic components.

The megaTAL technology uses customizable enzymes designed to recognize and cut specific DNA regions, while the mRNA (messenger RNA) platform allows the delivery of mRNA — the molecule generated from DNA that serves as a template for protein production — to cells.

Novo Nordisk is in charge of funding all research and development activities, with the option to exclusively license 2seventy bio’s megaTAL technology and gene-editing technology for the treatment of hemophilia A.

2seventy bio also has entered into a partnership with Genevant Sciences to gain access to its lipid nanoparticle system, which is intended to allow megaTAL mRNA to be effectively delivered to cells in the liver where FVIII is produced.