Live biotherapeutic products (LBPs) are a promising, newly emergent class of therapeutic agents that utilize live microorganisms, such as bacteria, viruses, or fungi, to prevent or treat various diseases. Depending on LBP type, disease target and route of administration, LBPs exert their therapeutic effects through various mechanisms in complex and manifold ways — via modulation of the gut microbiota, regulation of immune responses, production of antimicrobial substances, and enhancement of barrier functions in the body after engraftment. Clinical applications of LBPs include gastrointestinal disorders (e.g., inflammatory bowel disease [IBD] and irritable bowel syndrome [IBS]), metabolic disorders (e.g., obesity and diabetes), infectious diseases (e.g., recurrent Clostridioides difficile infection [rCDI]), mental health conditions (e.g., depression and anxiety), and certain cancers. Ongoing challenges include optimal standardization during LBP formulation (e.g., stability and viability of live microorganisms during manufacturing, storage, and delivery), establishing optimal dosing regimens, understanding the complex interactions between LBPs and tissue-specific human microbiota, and addressing potential safety signals following transplantation.

The regulatory landscape for LBPs is still evolving with the development of new guidelines outlining requirements for preclinical and clinical studies, manufacturing processes, quality control, and post-marketing surveillance. In 2012, the US Food and Drug Administration (FDA) published the first guidance for LBP development, which were defined as biological products that: 1) contain live organisms; 2) are applicable to the treatment, prevention or cure of a disease, and; 3) are not a vaccine. Eleven years later, the first two LBPs have been approved by the US FDA for commercialization.

This research topic aims to publish the latest advances in LBP development, clinical evaluation and outcomes assessment to ultimately improve LBP awareness and demonstrate how LBPs can revolutionize disease treatment.

Submission of original research involving clinical data, reviews and minireviews, methods, resources, and perspectives on future directions and challenges are welcome. Potential topics may include, but are not limited to:

• Exploring factors impacting regulatory decision-making and the status of US-, EU- and region-specific regulatory guidance regarding LBP use

• Developing improved approaches to LBP manufacturing and standardization

• Gaining a better appreciation for the diversity and complexity of LBP mechanisms of action

• Optimizing clinical trial methodology, data generation, biostatistics, and sequencing validation/standardization

• Evaluating real-world evidence generation and the development of biomarkers assessing LBP impact, disease risk and monitoring

• Exploring emerging therapy areas of interest, including reproductive and women’s health, rCDI, IBD, IBS, immuno-oncology, metabolic disease, liver disease, autism and the gut-brain axis

• Investigating novel aspects of LBP-based treatment modalities, including alignment on engraftment, decolonization, and antimicrobial resistance

• Probing unique perspectives on traditional drug development concepts and their application to LBPs

• Evaluating new approaches to health economics and outcomes assessments and patient perspectives on LBPs and the medical value of disease-specific unmet needs

• Integrating clinical evidence with medical value in the context of the investment landscape

The Topic Editor Ken Blount is employed by Ferring Pharmaceuticals. The Topic Editor Christine Lee received financial support from Ferring Pharmaceuticals, Seres Therapeutics, and Summit Therapeutics; and the Topic Editor Sahil Khanna received financial support from Ferring Pharmaceuticals, Seres Therapeutics, Vedanta, Finch and Pfizer. The other Topic Editors declare no competing interests with regard to the Research Topic subject.

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Frontiers in Microbiomes

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