Genevant, Novo Nordisk partner on gene-editing treatment for hem A
Partners to use proprietary platform for 'potentially transformative' therapy
Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology.
The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete a DNA sequence — for the bleeding disorder.
“We are thrilled to work with Novo Nordisk in the development of a potentially transformative gene editing treatment for a patient population in need of novel therapeutic approaches,” Pete Lutwyche, PhD, Genevant’s president and CEO, said in a company press release.
“This important collaboration highlights the role that state-of-the-art LNP delivery technology can play in harnessing the massive potential of RNA-based medicines,” Lutwyche said.
New collaboration builds on research and development partnership
The nonexclusive license agreement builds upon a research and development collaboration between the pharmaceutical company Novo Nordisk and the biotechnology company 2seventy bio to develop gene therapy candidates for the blood-clotting disorder.
Under that agreement, an option was exercised to exclusively license 2seventy Bio’s megaTAL technology — customizable enzymes crafted to recognize and cut certain DNA regions — to Novo Nordisk. Financial terms of the agreement between Genevant and Denmark-based Novo Nordisk are in accordance with the option agreement announced in January 2022 between Genevant and 2seventy bio.
When announced, the venture between 2seventyBio and Novo Nordisk expanded upon a partnership between Bluebird Bio — 2seventy’s parent company — and Novo Nordisk to find new gene therapy candidates for hemophilia and other disorders.
2seventy bio’s approach could potentially be used to correct the faulty F8 gene that causes hemophilia A and restore the body’s ability to produce a functional version of factor VIII (FVIII), the blood clotting protein that’s missing or is defective in people with the disorder.
A key preclinical milestone in the joint program with Novo Nordisk to develop a gene-editing tool for hemophilia A was announced by 2seventy bio in May. At that time, details of the proof-of-concept study that met the milestone were undisclosed.
2seventy bio’s separate partnership with Genevant Sciences seeks to leverage Genevant’s lipid nanoparticle system to effectively transport and deliver megaTAL mRNA to liver cells where FVIII is produced.
We recognize the importance of delivery to the success of RNA therapeutics and are excited to now be working directly together with Genevant to bring a next-generation gene editing therapeutic to individuals living with hemophilia A.
With its latest partnership, Genevant — a nucleic acid delivery company with an expansive LNP patent portfolio — seeks to work with Novo Nordisk to develop an in vivo or in the body gene-editing therapy for hemophilia A. In this type of treatment, gene-editing tools are directly infused into patients to genetically modify specific cells.
“We recognize the importance of delivery to the success of RNA therapeutics and are excited to now be working directly together with Genevant to bring a next-generation gene editing therapeutic to individuals living with hemophilia A,” said Karina Thorn, corporate vice president of RNA and gene therapies at Novo Nordisk.