Moderna is keeping its foot on the gas in 2023 when it comes to expanding beyond mRNA. Having signed a gene-editing pact in January and scooped up a DNA manufacturer in February, this month sees the biotech giant hand over $76 million as part of a collaboration centered on nonviral genetic medicines with Generation Bio.
On one side of the deal, Moderna has the option to license two immune cell programs that will be jointly developed based on Generation Bio’s stealth cell-targeted lipid nanoparticles to deliver closed-ended DNA. Alongside this, Moderna also has an option to two liver-focused programs, which will use Generation Bio’s liver-targeted lipid nanoparticles. Finally, Moderna retains an option to license a third program for either immune cells or the liver.
In return, Moderna is handing over $40 million in upfront cash and $36 million as an equity investment in Generation. Moderna will bankroll all R&D work, including a research prepayment for Generation, which in turn will be eligible for milestones and royalties of all resulting therapies. Moderna will also get the right to buy more shares in Generation with a future equity financing.
In the longer-term, the two companies plan to “leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines,” according to the Thursday morning press release. Again, Generation will be eligible to milestone and royalties from any resulting products that use its lipid nanoparticle technology.
Generation touts its closed-ended DNA as having almost three times the capacity of AAV capsids, enabling it to carry large or multiple genes to a cell. By delivering this DNA via its cell-targeted lipid nanoparticle delivery system, the Cambridge, Mass.-based biotech aims not only to deliver these large genetic payloads to specific tissues and cell types but also to make it easier to titrate and redose to adjust or extend gene expression levels in individual patients.
“Through this collaboration, which builds on Generation Bio’s nonviral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement,” Rose Loughlin, Ph.D., Moderna's senior vice president for research and early development, said in the release. “We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients.”
“Nonviral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale,” said Generation’s Chief Strategy Officer Phillip Samayoa, Ph.D. “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.”
While making its name in COVID-19 and working on a stable of vaccines in other respiratory disease, as well as cancer, Moderna has also been exploring a range of genetic medicine avenues in recent months. It opened the year making its first-ever acquisition in the form of Japanese DNA manufacturer OriCiro, before teaming up with ElevateBio’s Life Edit Therapeutics with the aim of developing next-gen gene-editing therapies for hard-to-treat diseases.
While Generation has yet to bring a candidate into the clinic, it is working on a spread of programs in various liver and retina-related conditions. The Moderna deal marks a rare collaboration for Generation, which last teamed up with a pharma when it allied with Vir Biotechnology in the early days of the pandemic.
Investors seemed happy with the deal, sending Generation's shares up over 27.9% to $5.00 in the opening hour of trading on Thursday morning from a Wednesday closing price of $3.91.