驾驭多样性：描绘微生物组治疗的未来，来自微生物组前沿的声音

Harnessing the Multitudes:Charting the Future ofMicrobiome Therapeutics Voices from the microbiome frontier STEFAN LUTZMAYER, Senior Consultant, EMEA Thought Leadership, IQVIAISMA HACHI, Senior Principal, Consulting Services, IQVIAISABELLE DE CREMOUX, CEO and Managing Partner, Seventure PartnersTOMAS DE WOUTERS, CEO & Co-founder, PharmaBiome Table of contents Abstract1Introduction — quovadis microbiome1Microbiome innovation2Clinical and translationalprogress3Collaboration and regulatory alignment7Navigating investor caution to unlock microbiomeopportunities8Harnessing the potential of the microbiome: Key success factors for advancinginnovation 10Conclusion13Acknowledgments13About the authors14References15 Abstract The FDA approved the first FMT-based microbiome drugs developed byFerringPharmaceuticals and Seres Therapeutics in November 2022 and April2023 respectively. This milestone sent waves of enthusiasm through theentire field who aims to bring the human microbiome and its modulationas an attractive modality into the spotlight. Two years later, it is time toreflect on the impact these approvals have had on patients and how, goingforward, companies want to harness the power of the microbiome for betterhealth outcomes. This paper consolidates industry insights and companyperspectives on the microbiome, providing a comprehensive overview oftrends in the microbiome field. It also provides an analysis of the challengesraised by microbiome stakeholders, as well as the key success factors shapingthe field. By addressing these challenges through a focus on broad therapeuticpotential and innovation, fostering ecosystem collaboration, buildingstakeholder trust, embracing patient-centric approaches, and pursuingregulatory clarity, the microbiome field is poised to overcome its obstacles andunlock its immense potential. This paper examines how the field is navigating thatcomplexity. Drawing on clinical and pipeline data,as well as interviews with leaders across biotech,investment, and regulation, we explore how companiesare redefining success, focusing pipelines, and buildingstakeholder trust. Their perspectives are integratedthroughout the analysis to ground our findings in therealities of development and commercialisation. Introduction — quovadis microbiome As the microbiome therapeutics field matures,companies, regulators, and investors face a sharedchallenge: how to turn scientific potential into scalableproducts, with credible health impact. Across theindustry, there is growing alignment around thetherapeutic potential of the microbiome. A wave ofscientific advances, clinical progress, and regulatoryrecognition is helping the field transition from earlyexploration to focused, evidence-based productdevelopment and validation. Companies are advancingfrom live bacterial consortia to targeted microbiomemodulation, supported by increasingly sophisticateddata analytics and translational models. At thesame time, efforts to harmonise clinical trial design,manufacturing standards, and regulatory pathways aregaining traction — laying the foundation forbroaderadoption and impact. Our focus is on microbiome-modulating therapiescurrently in development, including live biotherapeuticproducts, rather than the broader microbiome spaceof diagnostics, nutrition, or wellness products (Box 1).We aim to equip biopharma strategists, innovationleaders, and investors with a clear-eyed view of what’sworking, what’s stalling, and where the greatestopportunities now lie. Box 1: Mapping microbiome therapeutics Microbiome-modulating therapies span a continuum — from minimally processed microbial communities tohighly defined biological products.1Each therapy must be assessed based on its degree of characterisation,control, and intended use. Faecal microbial transplantation (FMT) Minimally processed stool-derived material, typically from screened donors. Donor origin plays a major role in benefit-riskassessment. Often, made at the hospital, variable composition; limited characterisation and control. Microbiome ecosystem therapies Medical products prepared at industrial scale following reproducible industrial manufacturing process and according toconsistent quality, safety and efficacy standards. These products are of standardised consistency and are intended to bedistributed to patients on a large scale and, therefore, need marketing authorisation to be commercialised. Live biotherapeutic products (LBPs) Engineered or defined consortia of live microorganisms developed as drugs, typically for specific clinical indications.Regulated as biologics or drugs. Probiotics, prebiotics and postbiotics Found in food, supplements, or consumer health products. Generally, not intended to treat disease; regulated as food ordietary supplements. Non-living biotherapeutics and phage therapiesInclude inactivated microbes or bacteriophages. No live organisms; offer targeted action with full product control. See ourrecentblog post“The good