RARE Daily

Moderna and Generation Bio Enter Strategic Collaboration to Develop Non-Viral Genetic Medicines

March 23, 2023

Rare Daily Staff

Messenger RNA pioneer Moderna has entered into a strategic collaboration with genetic medicines developer Generation Bio to combine Moderna’s biological and technical expertise with core technologies of Generation Bio’s non-viral genetic medicine platform.

The collaboration aims to expand the application of each company’s platform by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines.

“Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients,” said Rose Loughlin, Moderna’s senior vice president for research and early development. “Through this collaboration, which builds on Generation Bio’s non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement.”

Under the terms of the agreement, Moderna may advance two immune cell programs, each of which may use a jointly developed cell-targeted lipid nanoparticle (ctLNP) to deliver high-capacity, engineered, closed-end DNA (ceDNA). In addition, Moderna may advance two liver programs, each of which may use a liver-targeted ctLNP developed by Generation Bio to deliver ceDNA. Moderna retains an option to license a third program for either immune cells or the liver.

Generation Bio will receive a $40 million upfront cash payment and a $36 million equity investment issued at a premium over recent share prices. Moderna will fund all collaboration work, including a research pre-payment. Generation Bio is also eligible for future development, regulatory and commercial milestone payments, as well as royalties on global net sales of liver-targeted and immune cell-targeted products commercialized under the agreement.

The agreement additionally provides Moderna with the right, subject to certain terms and conditions, to purchase additional shares of common stock in connection with a future equity financing by Generation Bio.

Moderna and Generation Bio will both leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines, with downstream economics on products utilizing such technology. Generation Bio is eligible to receive certain exclusivity fees as well as potential development and regulatory milestones and royalties on products that Moderna advances using ctLNP technology developed under the collaboration.

“Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale,” said Phillip Samayoa, chief strategy officer of Generation Bio. “This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies.”

Photo: Phillip Samayoa, chief strategy officer of Generation Bio

Stay Connected

Sign up for updates straight to your inbox.