April 19, 2019

Current Status and Initial Considerations for Successful Development and Commercialization of Microbiome Therapies

By: Thunicia Moodley and Erin Mistry


Research in the area of live biotherapeutics has exploded in the last seven years. Even so, there is still much to learn about whether a dysregulated gut microbiome causes disease, or whether disease leads to dysbiosis. Dysbiosis has been implicated in multiple therapy areas including gastrointestinal disorders, immunology, neurology and oncology, among others. In addition, the therapeutic effects of several pharmaceuticals have been shown to be mediated by the gut microbiome.

Despite the scientific questions that remain to be investigated, several biotechnology companies have emerged with a focus on testing various approaches to developing therapies targeting the gut microbiome. Large, established pharmaceutical companies have also recognized the potential of live biotherapeutics and are investing in partnerships with these companies—or, as in the case of at least one company, are establishing an entire institute dedicated to the development of live biotherapeutics.

Although three therapies have reached Phase III development so far, they face numerous challenges in reaching the market—e.g., achieving regulatory approval, determining an appropriate price and obtaining reimbursement, and overcoming barriers to adoption of live biotherapeutics. Our paper provides a summary of the current gut microbiome therapy development landscape and an overview of the commercialization challenges faced.

The Microbiome: A Closer Look

The microbiome is an ecosystem of bacteria. Bacteria exist in the environment as well as in or on many parts of our bodies, including on our skin and in our eyes and gut. The gut microbiome, specifically, is also known as the “second brain” or “second genome”—the names alluding to the importance of the gut microbiome to our health. There are approximately 10 trillion bacterial cells on the human body, 80 percent of which are beneficial. The microbiome is assembled at birth, develops with the host and is influenced by environmental factors such as diet and antibiotics. Recently, a role for human genetic variation has emerged as also being influential in accounting for interpersonal differences in microbiomes.


An explosion in basic and applied biomedical research

Understanding of the microbiome and its relationship to health and disease has grown exponentially over the past decade. The Human Microbiome Project, a five-year project initiated by the National Institutes of Health (NIH), was conducted to characterize the microbial communities at five major body sites and to create a baseline database for future health and disease research connected to microbiomes. Federal investments in microbiome research tripled from 2012 to 2014, totaling more than $922 million during that period, according to a report from the National Science and Technology Council. In 2016, the National Microbiome Initiative was launched with $121 million of funding to support research on the microorganisms that live in or on the human body, plants and other ecosystems and to provide a better understanding of their role in human and environmental health. In 2018, a group of 23 U.S. government agencies, including the National Science Foundation (NSF), announced they have joined to produce a five-year Interagency Strategic Plan for Microbiome Research, which outlines the objectives, structure and principles for coordinated microbiome research.