mRNA-based therapeutics pioneer innovation in the biopharmaceutical industry

By Andre Rickard, The Science Advisory Board contributing writer

February 14, 2022 -- In the age of COVID-19, many novel therapeutics have been brought to the forefront, most notably mRNA-based therapies. This technology has been used in two of the most prominent COVID-19 vaccines currently available in the U.S. under the U.S. Food and Drug Administration emergency use authorization. As a result, some companies are expanding mRNA-based technology to apply it to other therapies while further strengthening its use in vaccines.

BioNTech, Novo Nordisk, and Pfizer are a few of the leaders in this innovative field of therapeutics, according to Cell and Gene Therapy Business Outlook, a sister publication of ScienceBoard.

BioNTech and Crescendo Biologics

BioNTech is a biotechnology company based in Mainz, Germany, with platforms focused on mRNA therapeutics, engineered cell therapies, antibodies, and small molecule immunomodulators. In addition to the company's collaboration with Pfizer on its COVID-19 vaccine, BioNTech focuses much of its efforts specifically on the development of cancer treatments using its cutting-edge technologies as immunotherapies.

In support of these efforts, BioNTech has collaborated with Crescendo Biologics to develop precision immunotherapies against cancer in addition to other undisclosed diseases. As part of this collaboration, Crescendo will receive $40 million upfront, as well as development, regulatory, and commercial milestone payments potentially totaling up to $750 million.

In turn, Crescendo will contribute its Humabody heavy chain-variable domain platform to deliver fully human mRNA-based antibody domains and engineered cell therapies to targets nominated by BioNTech.

Additional financial details of this collaboration were not disclosed.

Novo Nordisk and 2seventy bio

Novo Nordisk is a leading global healthcare company headquartered in Denmark driven to develop treatments for serious chronic diseases, including diabetes, obesity, endocrine disorders, and other serious chronic diseases. In addition, the company develops treatments for rare blood disorders, such as hemophilia A.

To further the treatment options for hemophilia A, Novo Nordisk has entered into a license agreement with 2seventy bio for R&D of an in vivo gene editing treatment. Through this agreement, Novo Nordisk will utilize 2seventy bio's mRNA-based megaTAL technology to develop a treatment using this novel therapeutic approach. This collaboration represents a significant shift in the application of mRNA-based therapeutics.

2seventy bio will receive an upfront payment of $5 million in addition to near-term milestone and option exercise payments of up to $35 million. 2seventy bio will also be eligible for further milestone payments and royalties.

Additional financial details of the collaboration were not disclosed.

Pfizer and Codex DNA

Pfizer is a leading research-based biopharmaceutical company offering treatments for a variety of diseases, ranging from cardiovascular disease to common childhood illnesses. As a partner in the development of a leading mRNA-based COVID-19 vaccine with BioNTech, Pfizer continues to collaborate with other companies to build on its innovations. One such collaboration includes Pfizer's partnership and licensing agreement with Codex DNA.

Through the agreement with Codex, Pfizer will be able to access and further develop Codex DNA's novel enzymatic DNA synthesis (EDS) technology for potential application to Pfizer's existing mRNA-based vaccines plus other biopharmaceutical products. Codex DNA's EDS technology is a scalable and cost-effective research approach designed to reduce the timelines for constructing synthetic DNA, RNA, and protein.

By partnering with Pfizer, Codex's technology could be used for more efficient and effective R&D of mRNA-based vaccines, such as the COVID-19 vaccine. The financial details of the collaboration were not disclosed.

As unprecedented diseases like COVID-19 continue to present themselves, new technologies are essential in the development of vaccines and treatments. The widespread adoption of mRNA technology in COVID-19 vaccines demonstrates this point clearly.

Companies like BioNTech, Novo Nordisk, and Pfizer have recognized this growing need and are expanding on such technologies by collaborating and forging partnerships with other healthcare companies. As more companies follow suit with the innovations and partnerships of their own, the healthcare field stands a greater chance of conquering novel diseases alongside existing diseases.

To stay up to date on the latest business happenings in the cell and gene therapy industry, check out Cell and Gene Therapy Business Outlook today!

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