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Moderna, Biotech Partner on Non-Viral Genetic Drug Delivery

Gene therapy graphic

(Genome Research Limited, Flickr)

23 Mar. 2023. Two biotechnology companies are collaborating on creating genetic therapies delivered with lipid nanoscale particles using non-viral mechanisms to reach target cells. The research and licensing deal is expected to bring Generation Bio Co. in Cambridge, Massachusetts at least $76 million from Moderna Inc., also in Cambridge, with few other financial details disclosed.

Generation Bio is a seven year-old company developing alternatives to current methods for delivering genetic therapeutics with engineered benign viruses, most often adeno-associated viruses. For most people, adeno-associated viruses deliver genetic payloads by infecting target cells, but rarely cause disease or a serious immune response. However, these viruses carry only small amounts of genetic material and while benign to most people, may not be appropriate for people at greater risk of a harmful immune response.

The Generation Bio technology uses a synthetic nucleic acid it calls closed-end DNA that the company says is designed for gene therapy payloads. The company says its manufacturing process can quickly produce closed-end DNA to provide larger and longer-lasting genetic quantities, including multiple genes, in a single payload. Plus, says Generation Bio, those payloads are delivered to cells or tissue, in concentrations and doses appropriate for patient populations, with lipid or natural oil nanoparticles designed to reach specific cell targets.

Moderna emerged in the past few years as a leading developer of vaccines and therapies using synthetic messenger ribonucleic acid or mRNA to invoke immune responses in recipients. In most of Moderna’s programs, the company uses lipid nanoparticles for mRNA payload delivery. While the company is best known for its Covid-19 vaccines, Moderna is also developing vaccines for other infectious diseases, as well as cancer immunotherapies, treatments for autoimmune disorders, and inherited disease therapies. (Science & Enterprise first reported on then start-up Moderna Therapeutics in Dec. 2012, raising early venture funds.)

“Diverse nucleic acid cargos”

In their collaboration, the companies are developing two therapies provided by Moderna targeting immune-system cells, with cell-targeted lipid nanoparticles delivering closed-end DNA from Generation Bio. The companies may also partner on two other Moderna therapies targeting liver cells, with Moderna retaining an option to license another therapy for immune-system or liver cells. In addition, Moderna and Generation Bio may also collaborate on what they call a new generation of gene therapies that target immune-system cells operating in vivo or inside the body.

“Through this collaboration, which builds on Generation Bio’s non-viral genetic medicines platform,” says Moderna’s senior vice-president Rose Loughlin in a company statement, “we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement.”

Moderna is providing Generation Bio with an initial payment of $40 million, and taking a $36 million equity stake in Generation Bio. In addition, Moderna is funding all research work in the collaboration, with Generation Bio eligible for development, regulatory, and commercial milestone payments and royalties on future product sales, although dollar amounts of those payments are not disclosed. Moderna also has an option to purchase further Generation Bio shares.

“This collaboration represents a foundational investment in our platform science,” notes Generation Bio’s chief strategy officer Phillip Samayoa, “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.”

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