Why Bioplatforms Matter, and Can Even Save the World

What the bioplatform affords is not only the ability to generate multiple related or unrelated products in parallel to tackle a wide array of problems and markets, but also the flexibility to rapidly activate new initiatives in response to emerging opportunities and threats.

Take for instance Moderna, and its messenger RNA (mRNA) therapeutic bioplatform. It was devised, designed and developed to create an mRNA molecule to encode virtually any therapeutic protein, for use as a medicine or a vaccine. The company had developed the core, universal (meaning application-agnostic) capability to design, make and test mRNAs, and has been applying it to the development of multiple vaccines (including against cytomegalovirus, Zika, respiratory syncytial virus, influenza, and others), antiviral antibodies, cancer drugs, and rare disease enzymes. Already, the bioplatform’s value becomes imminently apparent in the sheer number and diversity of programs that a relatively small company like Moderna can pursue in parallel. Even more striking and consequential was the company’s ability to rapidly direct the bioplatform to the creation of novel proteins, such as the SARS-CoV-2 spike protein, within just two days of the protein’s sequence being released. The company and platform did not have to be drastically modified to tackle the pandemic, beyond having to dramatically scale up to meet the manufacturing and fill-finish demands of supplying countries around the globe with hundreds of millions of doses, exemplifying the true power of a genuinely universal platform.

Compare this to large pharmaceutical organizations that aren’t based on bioplatforms, but pride themselves on being vertically integrated companies spanning biological discovery to commercialization. Few if any were able to respond as quickly as Moderna to the pandemic, and those that did actually partnered with bioplatform companies to instantiate their vaccines or therapies.

An additional and important benefit of bioplatforms is the ability to categorize their application space into sub-modalities (categories of use cases), whereby success with a couple of examples in that category dramatically and predictably unlocks the likelihood of success with other products in that category. This is why the scientists at Moderna and the National Institutes of Allergy and Infectious Disease(NIAID) were confident that an mRNA-based vaccine to a newly discovered viral protein such as the SARS-CoV-2 spike protein would very likely work; prior preclinical and clinical successes with Moderna’s Zika and cytomegalovirus mRNA vaccines, indicated that they could rely on animal models and virus neutralization experiments to predict the efficacy and safety of any new mRNA vaccine they designed.

This ability to gain confidence in large application categories is not as easily afforded by small molecule or traditional protein-based therapeutics. In those drug classes, success with a small molecule against a given target indeed tells you that drugging that target is possible, but doesn’t dramatically improve the likelihood of success or speed when tackling the next new target (only slightly if the target is of the same ilk, such as with kinase targets, for example). Bioplatforms thereby render discontinuous problem landscapes into more continuous and easily navigable terrains, facilitating lateral moves once a beachhead has been established in a given application area.

Interestingly and excitingly, our continued understanding and mastery of biology is yielding a plethora of new bioplatforms, including nucleic acid-based, cell-based, and computationally-designed modalities that can produce smarter, more rationally-designed medicines, more quickly. Where the dual bioplatform tenets of application breadth and universality afford us limitless possibilities to tackle virtually any biological problem. This is propelling us into a golden age of biology and medicine, in which we are less and less reliant on serendipity for finding cures, and are now capable of engineering and generating creative solutions to help us all live longer, better and more productive lives.