Moderna, Inc., a biotechnology company that invented messenger RNA (mRNA) therapeutics and vaccines, and Generation Bio Co., a biotechnology company developing genetic medicines for people with common and rare diseases, recently announced that they have formed a strategic partnership to combine Moderna’s biological and technical expertise with the foundational technologies of Generation Bio’s non-viral genetic medicine platform. By creating innovative nucleic acid treatments, such as those that can reach immune cells, the alliance hopes to broaden the applications of each company’s platform and speed up the development of their individual pipelines of non-viral genetic medicines.
Rose Loughlin, Ph.D., Moderna’s senior vice president for research and early development, stated that the company “continues to invest in breakthrough technology to enable us to develop a range of revolutionary medicines for patients.” “With this partnership, which expands on Generation Bio’s non-viral genetic medicine technology, we have the ability to target immune cells with various nucleic acid cargos and the liver for gene replacement. As we continue to expand our therapeutic pipeline and bring nucleic acid medicines to a wider patient population, we are thrilled to have Generation Bio as a partner.”
According to Phillip Samayoa, Ph.D., chief strategy officer of Generation Bio, “Non-viral DNA treatments may give lasting, redosable, titratable genetic medicines to patients suffering from rare and common diseases on a worldwide scale.” “This partnership marks a crucial investment in our platform science, broadening our pipeline of initiatives for rare and common liver diseases beyond haemophilia A and advancing our efforts to use nucleic acid therapeutics to treat organs other than the liver. We are excited to work with Moderna to provide unique targeting for our stealth ctLNPs to reach immune cells, extending genetic therapeutics to new tissues and cell types.”
A jointly created ctLNP may be used to deliver ceDNA in each of the two immune cell projects that Moderna is allowed to progress under the conditions of the agreement. Furthermore, Moderna may pursue two liver programmes, each of which may deliver ceDNA using a liver-targeted ctLNP created by Generation Bio. Moderna still has the opportunity to licence a third software for the liver or immune cells. A $40 million upfront cash payment and a $36 million equity investment provided at a premium above previous share prices will be given to Generation Bio. Any collaborative activity, including a prepayment for research, will be funded by Moderna. Besides from royalties on worldwide net sales of liver- and immune cell-targeted therapies marketed under the agreement, Generation Bio is also eligible for future development, regulatory, and commercial milestone payments. In addition, the agreement gives Moderna the opportunity to buy extra shares of Common Stock in conjunction with a future Equity Financing by Generation Bio, subject to specified terms and conditions.
Furthermore, in vivo immune cell targeting as a new class of genetic therapies will be advanced by Moderna and Generation Bio together with downstream economics on products leveraging such technology. In addition to potential development and regulatory milestones, royalties, and certain exclusivity fees that Generation Bio may be entitled to collect, Moderna may advance products employing the ctLNP technology created as part of the agreement. Since its inception more than ten years ago, Moderna has evolved from a research-stage company advancing programmes in the field of messenger RNA (mRNA) to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a wide-ranging intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that enables quick clinical and commercial production at scale.
Genetic therapies are being developed by Generation Bio to offer enduring, repeatable treatments to patients suffering from both common and rare ailments. A novel DNA construct known as closed-ended DNA, or ceDNA, a special cell-targeted lipid nanoparticle delivery system, or ctLNP, and a highly scalable capsid-free manufacturing process that uses a patented cell-free rapid enzymatic synthesis, or RES, to produce ceDNA are all included in the company’s non-viral genetic medicine platform.
