abstracts & speaker proﬁles - pharmabiotics event · 2019-02-14 · abstracts & speaker...

speaker profiles & abstracts

Abstracts & Speaker Profiles

Key Note

TRANSLATING THE MICROBIOME IN THE ERA OF PRECISION MEDICINE

Abstract: The Alkek Center for Metagenomics and Microbiome Research (CMMR) at Baylor College of Medicine is pursuing numerous research and development e�orts in the study of how the microbiome impacts human disease, including autoimmune disease, neurodevelopmental disorders, cancer, and type 1 diabetes. Among these e�orts are the benchmarking and validation of protocols and strategies to engage large, complex cohorts and unique sample sources for metagenomic analyses delivered for academia and industry with our corporate partner, Diversigen. In addition, we have made a significant investment in preclinical model systems facilitate host-microbe studies and connect association to causality, as this is a critical hurdle for any microbiome research program. Among these models are a large germ free rodent facility and physiologically active human culture systems that mimic host susceptibility and response to determine microbe-host interactions of mucosal diseases. By implementing ‘omics-based approaches with these translational models, we are beginning to reveal host responses to microbial colonization and infection which can inform precision medicine-based targets for the detection and treatment of disease.

Dr. Petrosino is the Cullen Foundation endowed interim-chairman of the Department of Molecular Virology and Microbiology at Baylor College of Medicine (BCM). He is also Founder and Chief Scientific O�cer for Diversigen, Inc. Subsequent to his leadership role in the Human Microbiome Project, Dr. Petrosino launched the Alkek Center for Metagenomics and Microbiome Research (CMMR) in 2011 to catalyze translational microbiome research with clinicians and investigators from around the world. Together, CMMR and Diversigen have engaged in more than 400 diverse microbiome studies with over 200 collaborating groups internationally, leading to more than 130 authored contributions.

Joseph Petrosino | Baylor College of Medicine

DEVELOPMENT OF SYNTHETIC BIOTIC MEDICINESTM FOR PATIENTS

Abstract: Synlogic is pioneering the development of a novel class of living medicines, Synthetic Biotic medicines, based on its proprietary drug development platform. Synlogic leverages the tools and principles of synthetic biology to genetically engineer beneficial microbes to perform or deliver critical functions missing or damaged due to disease. Synthetic Biotic medicines are designed to act locally and have a systemic e�ect to address disease in patients. Synlogic’s two lead programs, SYNB1020 and SYNB1618, are orally administered and target hyperammonemia as a result of liver damage or genetic disease, and phenylketonuria, respectively. Synlogic is also developing SYNB1891 as an intratumorally administered Synthetic Biotic medicine for the treatment of cancer. In addition, the company is leveraging the broad potential of its


Maximising E�ciency in the LBP Development Process

UNDERSTANDING THE FUNDAMENTAL MECHANISMS UNDERLYING SIGNAL TRANSDUCTION BETWEEN MICROBIAL SPECIES

Abstract: Although the gut microbiota has been studied intensively for the last decades, the understanding of the fundamental mechanisms underlying signal transduction between the microbial species is still in its infancy. In this lecture, results will be shown that explain these fundamental signalling pathways from a first order perspective using elementary graph (network) theory. Using this approach, the mechanistic rationale underlying the empiric notion that a higher microbiota richness is linked to a more robustness can be elucidated. Moreover, recent results provide ingredients to rationally design fecal transplant analogues, also providing a mechanistic bridge from probiotic single strain to multi-strain preparations.

platform to create additional Synthetic Biotic medicines for the treatment of liver disease, as well as inflammatory and immune disorders, including Synlogic’s collaboration with AbbVie to develop Synthetic Biotic-based treatments for inflammatory bowel disease (IBD). This presentation will provide an overview of the Company’s strategy and progress to date in achieving the vision of developing new treatments for patients with unmet medical needs.

Aoife is currently President and CEO at Synlogic Inc. (Nasdaq:SYBX), a clinical stage biotechnology company applying synthetic biology to probiotics to develop novel, living medicines. She joined Synlogic as Chief Medical O�cer in September 2016, served as interim CEO from April 2018 and was promoted to the permanent position in October 2018. Previously she was Vice President and Head of the Rare Disease Innovation Unit at Biogen where her work resulted in the approval of ALPROLIXTM, ELOCTATETM and SPINRAZATM as well as the advancement of several early-phase programs and external collaborations. Aoife holds a medical degree from Trinity College Dublin, Ireland and completed residency and fellowship training in general internal medicine and endocrinology. She has completed post-doctoral training in clinical research and metabolism at the Beth Israel Deaconess Medical Center in Boston, is a graduate of the Harvard Medical School Scholars in Clinical Science Program and has > 40 academic publications. She serves as a Director of Ra Pharmaceuticals (Nasdaq:RARX) and is a member of the advisory board of the Harvard Masters in Clinical Investigation Program.

Aoife Brennan | Synlogic

Olaf Larsen | Yakult / VU University

Olaf Larsen (1972) studied chemistry at the VU University Amsterdam and obtained a PhD in biophysics at the same university. Following postdoctoral research in New York City and Amsterdam, he continued his career within industry. Olaf worked


for ASML, TNO and as a consultant life sciences within various organizations. Since 2012 he is heading the science department at Yakult Nederland. Since 2016, he is also part time assistant professor the VU University focussing on the valorization of microbiota management.

KEEP UP TO DATE WITH GUIDELINES

Abstract: A lot of guidelines implicated in the LBPs development and manufacturing are currently in draft or just been published. Working on the changes before the publication is crucial: First to be ready, once applicable, second to mitigate any risk for the Business. Some “critical” of them are presented, focusing on few major topics.

European Pharmacopea - LBPs (Live Biotherapeutic for Human Use) – Draft March 2017- The requirement for LBPs regarding traceability of the strain from the Research, “Sterile” media and control of the Product are now clear.

Annex 1 – GMP- : Manufacture of Sterile Medicinal Products – Draft Dec 2017-Thinking that the Annex 1-GMP concerns only sterile products is a big mistake. The principles of this guideline should be led to control the contamination risk: What about the “Aseptic Simulation Process”?

EU n°536/2014 - : Good Manufacturing Practices for IMPs for Human use– Draft Dec 2017- No more any doubt on the regulatory requirements for IMP (Investigational Medicinal Products), such as Traceability of changes, Qualification and the Release Chain.

Frederique acquired a multi-disciplinary vision of the Drug Development, from Research to Regulatory submission after more than 15 years experiences in Pharma/Biotech companies.After obtaining her Pharmacist graduation, she started in a French CDMO, producing and developing solid forms for commercial market. She passed the graduation “Engineer in Biotechnology” and became Head of QA - QP Deputy in a Swiss company for sterile products in clinical phases. Then, Frederique covered all the Pharmaceutical chain as Quality Director in a UK start-up developing LBPs.

Frederique Vieville | 5 Quality Business Development-Biotech

FLOW CYTOMETRY AS AN ALTERNATIVE METHOD FOR PROBIOTIC VIABILITY MEASUREMENT

Abstract: Viability of bacteria is traditionally determined by plating of samples on to agar media with enumeration of resultant colonies following growth. This methodology is slow, labour intensive and only enumerates bacteria based on their growth under specific environmental criteria. It is evident that an alternative rapid, high-throughput viability methodology is urgently required, especially for commercial probiotic cultures, probiotic supplements and foods into which probiotics are added. The probiotics


industry has responded to this need by employing Flow Cytometry (FCM). This methodology enables rapid analysis (< 5 mins for prepared stained samples) of individual bacterial cells enabling di�erentiation and enumeration of cultures into sub-populations of live, damaged or dead cells. Multi-parametric data is generated from FCM analysis of fluorescence generated from single cells stained based on their morphological or physiological properties. Using combinations of stains with FCM allows a unique insight into probiotic cell viability and vitality (functionality). This presentation will review the evidence for FCM as an alternative for rapid bacterial viability determination and will focus on a novel IDF/ISO FCM-based method developed by the probiotics sector based on a tri-staining method. Overall, FCM o�ers an alternative rapid methodology for determination of viability and vitality of probiotic cultures.

Martin Wilkinson is Associate Professor in Food Microbiology at the University of Limerick, who has established a research programme in the application of flow cytometry to the study of food bacteria. This has led to an output of a significant number of peer-reviewed publications in the field with research funding secured from national and international agencies. Martin commenced his work on the development of cytometry methods to study the permeabilisation and enzyme-release properties of Lactic Acid Bacterial starter cultures. This research has expanded into other areas of food pathogens and spore forming bacteria with the use of cell sorting to gain an insight into microbial physiology during food processing and storage. Martin’s most recent publication is a landmark independent review commissioned and funded by the International Probiotics Association on the potential of flow cytometry as a method of measuring bacterial viability in probiotic products. Martin is also the editor of a seminal text on microbial flow cytometry published by Caister Academic Press in 2015.

Martin Wilkinson | University of Limerick

Understanding, Leveraging & Engineering the Human Virome

THE VIROME AND ITS POTENTIAL FOR SHAPING THE MIRCOBIOME

Abstract: Viruses are the most abundant members of the human microbiome, yet relatively little is known about their ecology or their role in this diverse ecosystem. Because the majority of the identifiable viruses in the microbiome are bacteriophages, they could have substantial impacts upon the ecology of their host bacterial communities. A substantial proportion of the bacteriophages in the human microbiome are highly persistent, which suggests that they have evolved a dynamic equilibrium with their cellular hosts. We have been working to uncover the ecology of viral communities on human body surfaces to understand whether their ecology provides clues into the interactions between bacteria and their viruses in the human body. We have identified separate ecological groups that are distinguishable by alpha diversity, beta diversity, taxonomy, and virus sharing across body surfaces. Those surfaces inhabited by diverse bacterial communities also have diverse viral communities. In contrast, surfaces


inhabited by few if any bacteria have low diversity viral communities. To decipher the role of phages in these communities, we have been developing chemostat culture systems that simulate the magnitude, diversity, and taxonomy of bacteria and their viruses in the distal colon. There is dysbiosis in bacterial communities in a number of human disease conditions. We plan to use these cultured microbial ecosystems to assess the role of phages as potential drivers of bacterial dysbiosis in human microbial communities.

Dr. Pride is originally from Nashville, Tennessee. He received his undergraduate degree in biology from Wake Forest University, his Ph.D. in Microbiology and Immunology from Vanderbilt University, and his M.D. at New York University. He is board certified in Internal Medicine, and received subspecialty training in Infectious Diseases at Stanford University. Dr. Pride serves as the Director of the Molecular Microbiology laboratory and the Associate Director of the Clinical Microbiology laboratory at UC San Diego Health. Dr. Pride's major interests are in developing diagnostic tests for infectious diseases, and in understanding the role of microbial communities in health and disease.

David T. Pride | UCSD

REGULATORY AND MANUFACTURING COMPLIANCE OF PHAGE THERAPY PRODUCTS

Abstract: Facing the emergence of di�cult-to-threat bacterial infections, the perspective of using the anti-bacterial potential of bacteriophages has re-gained a significant interest in many countries. In Western countries , bacteriophages are not of common use, and, in turns, do not have a specific regulatory framework, for both the use and the production of the phages.

From a pharmaceutical classification point of view, phages fall in the categories of anti-infectious products and of biological products, given the intended use and their live nature. In addition, the compliance to the Good Manufacture Practices (GMP) is a requirement for any medicinal product.

During the EU-funded PhagoBurn’s project, we solved the challenges of 1) producing two cocktails of lytic bacteriophages in compliance with the GMPs, 2) performing a phase I-II clinical trial on burn patients su�ering from E. coli or P. aeruginosa infections.The rich interactions with the national authorities in 3 countries (France, Belgium, and Switzerland) during PhagoBurn’s experience, and in other phage-therapy projects, have shown the interest of the authorities to investigate the potential of phage therapy. Accordingly, the working frame for new development is better determined.

As head of R&D for Clean Cells, Laurent Bretaudeau contributed to the growth of the company by implementing innovative quality control methods and developing the manufacturing of biological products under Good Manufacturing Production (GMP) compliance. He supervised the GMP production of bacteriophages

Laurent Bretaudeau | LB4Biotech Consulting


for the phage therapy program PhagoBurn. During this European project, bacteriophages were used in a clinical trial with infected burn patients. Laurent had a major contribution in the regulatory process through the communication with the health authorities during the PhagoBurn project, and now in other projects based on phage therapy.

USING SYNTHETIC ENGINEERING TO DEVELOP NOVEL PHAGE THERAPIES FOR CHRONIC DISEASES

Abstract: BiomX is a microbiome drug discovery company developing customized phage therapies that seek and destroy harmful bacteria in chronic diseases such as IBD, liver disease and cancer. We discover and validate proprietary bacterial targets and customize our natural and engineered phage compositions against these targets. In this talk we will demonstration of synthetic biology capabilities to ‘reprogram’ phage in order to optimize their performance for therapeutic applications. Synthetic biology used include “reprogramming” of lysogenic (dormant) phage to a strictly lytic (active) mode, and the expansion of the phage host range to both achieve eradication of a wider array of bacterial strains and overcome bacterial resistance

1H-NMR METABOLOMIC ANALYSIS OF THE EFFECTS OF PROBIOTICS ADMINISTRATION ON HUMAN METABOLIC PHENOTYPE

Abstract: Establishing and preserving beneficial interactions between the host and its associated intestinal microbiota are key requirements for promoting health. Not surprisingly then, probiotics are increasingly being used to manipulate the composition of the gut microbiota and improving a healthy microbial community. But there’s more. The human metabolic phenotype could give us a very accurate and systemic representation of the health state, not only of the human organism, but, more correctly, of the ‘super organism’. The urine metabotype, besides being a snapshot of the host genotype, age, life-style and interaction with environmental factors, is also influenced by the gut microbiota. In this light the individual metabolic phenotype can be considered as a new dynamic mirror of the ‘superorganism’; how it functions and how it adapts. Their resilience could be defined as the system’s ability to recover from challenging conditions and return to health. The presentation will reveal findings of an ongoing study to extend our understanding of the human metabolome’s resilience and

Assaf Oron has served as chief business o�cer since January 2017. Prior to this position, he served for over a decade at Evogene (NYSE:EVGN), an agriculture biotechnology company which utilizes a proprietary integrated technology infrastructure to enhance seed traits underlying crop productivity. At Evogene, he worked in various roles such as executive vice president of corporate development and executive vice president of strategy and business development. Mr. Oron holds an M.Sc. in Biology (bioinformatics) and a B.Sc. in Chemistry and Economics, both from Tel Aviv University.

Assaf Oron | BiomX


how it responds to gut microflora modulation during probiotic treatment. He will show how it has been possible to monitor the molecular outcomes of probiotic administration on the human metabolism through a 1H-NMR-based metabolomic approach. The study – the first to look at the e�ects of probiotic administration through 1HNMR spectroscopy – shows that probiotics can influence people’s urine metabolic profiles without altering their individual metabotypes.

Marco holds a Master’s degree in microbiology and began his career as a researcher at Biolab laboratory in Novara, Italy, where he focused on research for the dietary supplement industry. At Probiotical he works with external innovation partners to drive new probiotic product development, supports worldwide sales and collaborates on research projects. He is a regular commentator on the technology, ef cacy and consumer bene ts of probiotic supplementation and healthy gut microbiota.

Marco Pane | Probiotical

OMICS

PROFILING MICROBIAL & PHAGE DIVERSITY BY ULTRA-HIGH-RESOLUTION MICROBIOMICS

Abstract: Ultra-high-resolution microbiomics can identify and profile microbes and phages with unprecedented precision and resolution. However, the increased resolution leads to higher perceived diversity and complexity. Here we flesh out the depth of this diversity and propose solutions to structure and manage the diversity. These structures can be used to map sub-species population structures, to capture accessory gene content and for making statistical associations with reduced multi-testing. Finally, we demonstrate how holistic approaches can be used to identify mechanistic associations between the microbiome and human health.

H. Bjørn Nielsen, PhD, Chief Scientific O�cer at Clinical-Microbiomics A/S, has since 2008 been a frontrunner in the field of microbiome research. His participation in the MetaHit project led to a series of important scientific papers describing the human gut microbiome, including his pioneering work on co-abundance binning of metagenomics data into metagenomic species, bacteriophages and other mobile genetic elements. Bjørn has authored 19 high-impact papers published in Nature and Cell journals, and his publications currently receive more than 2900 citations per year.

H. Bjørn Nielsen | Clinical Microbiomics

SHOTGUN SEQUENCING & STRAIN-LEVEL ANALYSIS: STUDY DESIGN FOR MICROBIOME R&D

Abstract: The emergence of Next-Generation Sequencing has changed the level of resolution that industry and academia can study the microbiome. As the industry transitions from 16S to Metagenomics, CosmosID is leveraging 10 years of R&D to educate and address the importance of standardization and reproducibility in metagenomics studies.

This presentation will outline the various applications of shotgun metagenomics at every step of the product development workflow; from early discovery through to clinical development and manufacturing QC. CosmosID will also present data on the impact that di�erent read-depths and lab methodologies can impact bioinformatics analysis along with recommendations on how to approach such studies.

A microbiologist and molecular biologist by training Arne Materna has received his doctoral degree from the University of Konstanz, Germany, and his postdoctoral training at the Massachusetts Institute of Technology. Dr. Materna later joined Pacific Biosciences as systems architect responsible for the system level modeling of a new Next-generation Sequencing device. In 2012 Dr. Materna joined CLC bio, acquired by QIAGEN in 2013. As Director for Genome Analysis Dr. Materna was business responsible for several segments in QIAGEN’s portfolio of bioinformatics solutions. Today Dr, Materna is part of CosmosID’s leadership team and directs the product management and marketing department.

Arne Materna | CosmosID


A HIGHER STANDARD FOR MICROBIOME STUDIES: ELEVATING STUDY DESIGN TO OPTIMISE DATA POTENTIAL

Abstract: Empowering the microbiome community to maximise discovery potential by focusing on developing enabling technology has always been at the core of DNA Genotek’s mission. We recently announced the merger of DNA Genotek and CoreBiome, creating a world-class solutions provider for multi-omics sampling, scalable processing and expert microbiome data analysis. This talk will illustrate how these new capabilities can help de-risk your microbiome discovery initiative and maximize your budget, without compromising quality. We will highlight a few examples of common challenges from study participant engagement, to processing and analysis, and how an optimised platform can help overcome them.

Heloise joined DNA Genotek in 2015 and has been a part of their microbiome team since 2016. She is currently responsible for new microbiome product development allowing her to use her years of experience working with researchers to better understand the market. Prior to joining DNA Genotek, she completed a PhD in applied bioscience at the University of Ontario Institute of Technology studying microbial communities of forensic samples.

Héloïse Breton | DNA Genotek Inc


STANDARDS FOR MICROBIOME STUDIES

Abstract: The human intestinal tract harbours a complex microbial ecosystem which plays a key role in nutrition and health. Interactions between food constituents, microbes and the host organism derive from a long co-evolution that resulted in a mutualistic association.

Current investigations into the human intestinal metagenome are delivering an extensive gene repertoire representative of its functional potentials. The most redundant genomic traits of the human intestinal microbiota are identified. These observations point towards the existence of discrete ecological arrangements known as enterotypes. It also shows a unique segregation of the human population into individuals with low versus high gene-counts. It not only gives an unprecedented view of the intestinal microbiota, but it also significantly expands our ability to look for specificities of the microbiota associated with human diseases and to ultimately validate microbial signatures of diagnostic and prognostic value in immune-mediated conditions.

The current view is that of an intimate symbiosis linking the microbiota and the host with marked implications in terms of ecological dynamics but also monitoring and modulation.

Both comparability and reproducibility are essential and yet delicate in metagenomic analysis of fecal samples. Lack of standards in the early steps of sampling and processing for the molecular profiling of human microbiomes has blurred the vision of disease associated alterations. Within the European Commission funded program IHMS, we have defined and proposed SOPs for sampling and processing of human fecal samples for metagenomics. We specifically quantified the e�ect of the DNA extraction process on the observed microbial composition, based on shotgun sequencing. We comparatively assessed 21 representative protocols implemented by participant laboratories. Among all factors including sequencing, sample storage and biological variability, DNA extraction had the highest impact on the results. We characterized biases of di�erent methods and assessed lab-to-lab transferability of the best methods. Finally, we propose a standardized sample handling and DNA extraction methodology for human fecal samples (www.microbiome-standards.org) that could serve as a benchmark to greatly improve comparability and consistency of human gut microbiome studies and facilitate meta-analyses.

Beyond analytical purposes, a similar need for standardization also applies to clinical microbiotherapy, where safety and stability are the highest concerns. Empirical observations in curing recurrent Clostridium di�cile infections indicate that eradication of the pathogen may be quite permissive in terms of ecological manipulation. Yet for other indications such as chronic conditions or harsh clinical interventions, the ultimate goal may be to fully master microbiome engineering for symbiosis restauration. This will require standardization of finely controlled microbiota processing workflows from collection to administration.

She has previous experience as an environmental microbiologist and is a member of the Canadian College of Microbiologists.


Joël is Research Director at INRA Micalis Institute “Food and Gut Microbiology for Human Health” (www.micalis.fr) and Scientific Director of MetaGenoPolis (www.mgps.eu), a pre-industrial demonstrator in quantitative and functional metagenomics of the national “Futures Investment” program. Gut microbial ecologist by training, Joel pioneered intestinal metagenomics towards food-microbe-host interactions as well as diagnostic applications. With > 30 years of academic research and > 200 publications, Joël aims to provide a better understanding of man-microbes symbiosis towards personalized preventive nutrition and precision medicine. Joel is co-founder and scientific advisor of www.maat-pharma.com. He chairs the www.gutmicrobiotaforhealth.com scientific web-platform.

Joël Dore | INRA

Advancing Applied Microbiome Research

PRACTICAL CHALLENGES IN MICROBIOME CLINICAL TRIALS

Abstract: Now is an exciting time in microbiome research as more products enter the clinical stages of development. We will discuss the practical challenges faced from the initial study design and start-up processes through to the recruitment for and running of the trials to achieve the desired outcomes.

Despite countless studies and applications made, the European Food Safety Authority has still yet to approve a single health claim for probiotics. We will examine why this is the case and what can be learned from this scenario. Whether there is an endpoint of a health claim or a medical claim for any microbiological product, the required standards must be achieved. Navigating the alphabet soup of EFSA, EMA, GCP, IMP, GMP, HAACP and so on, is not always a straightforward exercise. From getting the initial regulatory and ethical approvals to the design of recruitment campaigns, we will see how each of these aspects come into play and are addressed. The appropriate study design must also be considered in this process. For most clinical studies the critical factor which causes delay is patient recruitment. We will discuss recruitment methodology including social media and television advertising with detailed examples of enrolling both child and adult populations.

Finally, as we move toward the future of clinical trials, we will discuss the role of patient reported outcomes (PROs) and the conduct of “remote studies” in the microbiome field.

Alan Wade is a physician with over 30 years of clinical trial experience. He is an acknowledged expert in pharmaceutical development having acted as a consultant for many pharmaceutical companies advising on development pathways and clinical trial design. His company, CPS Research, has conducted pivotal studies in a wide spectrum of medical disorders including dermatology, gynaecology, cardiovascular, respiratory and CNS

Alan G. Wade | CPS Research


disorders. An expert in the treatment of CNS disorders, he has published widely in the established medical literature and lectured internationally on the role of drugs a�ecting mood disorders, melatonin in circadian rhythm disorders and the role of patient reported outcomes in future clinical trial design.

Lindsay is the Microbiome Research Leader at the Quadram Institute Bioscience (QIB) and a Wellcome Trust Investigator.Her lab’s research focus involves defining microbe-host interactions, with a focus on early life, and the genus Bifidobacterium.

Lindsay obtained a BSc in Microbiology from the University of Glasgow, a PhD in Microbiology and Immunology from the University of Cambridge (Wellcome Trust Sanger Institute), and was a postdoctoral fellow at University College Cork, Ireland (APC Microbiome Institute). She returned to the UK to take up a Senior Lectureship at the University of East Anglia before moving to QIB end of 2015.

Lindsay J. Hall | Quadram Institute Bioscience

MODULATING THE PRETERM GUT MICROBIOTA

Abstract: Initial colonisation of the gut by pioneer bacterial species is the first key step for host well-being, including immune defence development. The process of initial gut microbiota colonisation in preterm babies (1:9 live births defined as < 37 weeks gestation) is radically interrupted due to a variety of factors including mode of delivery and antibiotics. This aberrant colonisation of premature infants appears pivotal to the development of a number of diseases, including necrotising enterocolitis (NEC), which is linked to overgrowth of opportunistic pathogens. Importantly modulation of the preterm microbiota may aid in prevention of NEC in preterm infants. I will discuss how Bifidobacterium supplementation represents a powerful opportunity for strategically manipulating the wider early life microbiota when bacterial assembly is disturbed within the context of preterm birth, with correlations to health outcomes, and also discuss new approaches for rapid preterm microbiota profiling and diagnosis of pathogenic NEC-associated taxa.

UNLOCKING THE POTENTIAL OF THE MICROBIOME: OXALOBACTER FORMIGENES FOR THE TREATMENT OF PRIMARY HYPEROXALURIA

Abstract: Primary hyperoxaluria is a rare, inherited disease characterized by an overproduction of endogenous oxalate in the liver. Very high concentrations of blood and urinary oxalate lead to complications with kidney stones and calcification of the kidneys (nephrocalcinosis) early in life in Primary hyperoxaluria patients. Left untreated, the disease progresses with kidney deterioration and eventually into End-Stage Renal Disease (ESRD) requiring dialysis. Since removal of oxalate via dialysis is insu�cient to match the endogenous production, calcium oxalate accumulates also in other various tissues. The patients are subject for pre-mature death if not treated with a combined


liver-kidney transplantation.

OxThera pursues the development of Oxabact®, a live biotherapeutic drug made of lyophilized Oxalobacter formigenes, an anaerobe bacterium that solely relies on oxalate as its energy source. Oxabact® utilize a bimodal mode of action, where the bacteria emit signal peptides that both activate and enhance the excretion of oxalate from plasma into the intestinal lumen. The therapeutic potential of Oxabact® in Primary hyperoxaluria patients involves the reduction of oxalate concentrations in plasma and urine, thereby protecting the kidneys and potentially addressing the systemic build-up of oxalate in organs like the heart, bone and soft tissues.

OxThera conducted its first clinical trial with lyophilized live Oxalobacter formigenes in 2003. To date, OxThera has completed three double-blinded phase II/III clinical studies with di�erent formulations (OC3 and OC5) and are currently pursuing a fourth multicenter, international, double-blinded phase III clinical trial investigating the safety and e�cacy of the improved Oxabact® formulation, OC5 in Primary hyperoxaluria patients with maintained renal function. OxThera is currently also conducting a phase II, prospective, open-label study to investigate e�cacy and safety of Oxabact®, OC5, in patients with Primary hyperoxaluria that are treated under a stable dialysis regimen.

Pre-clinical and clinical studies have demonstrated functionality and safety of Oxabact® and has been found well tolerated by over 100 patients with Primary hyperoxaluria.

A 52-week interim analysis of the current phase II open-label study concluded that Oxabact® treatment is safe and that plasma oxalate concentrations were reduced during the treatment time in Primary hyperoxaluria patients on dialysis who did not change their dialysis regimen. These findings would be of a substantial clinical significance for clinical outcome and future transplantation outcome.

Confirmation of long-term e�cacy and safety is now underway through the ongoing phase III, double-blind, placebo-controlled study, where subjects are being randomized 1:1 to receive either OC5 or placebo, and are o�ered to pursue a long-term follow up study to prove clinical benefit

Maria Akerman is Director of Process R&D at OxThera with focus on Oxabact®, a live biotherapeutics based on Oxalobacter formigenes bacteria. Maria Akerman received her Ph.D. in Bioengineering from University of California, San Diego in 2004. She has worked with biologicals for 15 years in both large and small pharmaceutical companies and was recruited to OxThera in 2012. Process development resulted in an improved Oxabact® product, currently in use in clinical trials. Dr. Akerman’s expertise is in early, biological products where a solid CMC strategy and understanding of the drug’s mode of action is key to reliable clinical outcomes.

Maria Akerman | OxThera

Molecular microbiologist (PhD 1995). Invented and pioneered ActoBiotics technology (Science, 2000) which uses recombinant Lactococcus lactis for topical and active delivery of peptide bioactives (cytokines, antibodies, tolerigens). Invented a robust environmental containment system (Nature Biotechnology, 2003) which enabled the first clinical studies ever, using genetically modified bacteria as therapeutics. Holds 25 years of experience in bacterial genetic engineering and production technology, the use of live, genetically modified microbiota as therapeutics, environmental impact assessment in the context of deliberate release. Involved in the running of several clinical trials worldwide. Co-founder of ActoGeniX and currently Chief Technology O�cer at ActoBio Therapeutics, Belgium.

Lothar Steidler | ActoBio Therapeutics

ACTOBIOTICS®, LACTOCOCCUS-BASED BIOPHARMACEUTICALS FOR LOCAL DELIVERY OF PRECISE THERAPEUTICS AT TARGET SITES

Abstract: ACTOBIOTICS®, represent a unique delivery platform with demonstrated safety and tolerability, based on food derived Lactococcus lactis which are genetically engineered to produce precisetherapeutic proteins (cytokines, antibodies, tolerigens). These microbial compounds are precisely tailored for disease modification through e.g. anti-inflammatory or tolerance inducing activity and focus on areas with high unmet medical needs. A scalable and robust manufacturing process is in place and targeted delivery is achieved via oral capsule, oral rinse or topical solution.

TITLE TO BE CONFIRMED

Abstract: Coming Soon


WATCH THIS SPACE


Skin Microbiome Therapeutics: From Lab to Product

SKIN MICROBIOME AND RELATED DISEASES

Abstract: The human skin harbors a complex microbiota which composition changes based on the di�erent anatomical site. The skin microbiota is composed by both aerobic bacteria (staphylococci and streptococci) and anaerobic bacteria (propionibacteria), being directly involved in several severe skin diseases such as psoriasis, atopic dermatitis and acne. In the past Staphyloccus aureus, indeed, seemed to have a pivotal role in the progression of atopic dermatitis, but recently other microbial targets have been identified. Moreover, the high variability of skin microbiota due to lifestyle, sampling site and the kind of samples collected (swabs or skin biopsies) make the characterization of cutaneous microbiota a fascinating challenge for microbiologists. To date, metagenomic studies based on the combination of bacterial abundance and microbial networks are essential to clearly understand the specific role of cutaneous microorganisms, mainly considering a pathological condition. Psoriatic plaques, for instance, show a reduced biodiversity if compared to healthy skin and a change of microbial distribution in the microbiota, as di�erent bacterial hubs can be observed comparing psoriatic skin to healthy one. In conclusion, system biology is a fundamental approach to characterize the skin microbiota, considering both pathological conditions and the di�erent anatomical site under investigation.

Professor of Clinical Microbiology at University of Milan -Faculty of Medicine and Surgery – Dept of Biomedical Sciences for Health – Milan. Working Area Microbiology and Clinical Microbiology. Professor Drago is Directive Council Member of AMCLI and Responsible of the Microbioma Working Group in Italy. He is Past President of the International Society of Microbiota andCoordinator of WAIOT Scientific Commettee. The scientific interests are: microbiota and diseases, probiotics, antibiotics resistance and biofilm related infections.

Lorenzo Drago | University of Milan

HIGH RESOLUTION METAPROTEOMICS AND BIOINFORMATICS ANALYSES TO DECIPHER MICROBIOTA FUNCTIONS

Abstract: Functional studies of the skin microbial communities and the interactions among community members and with the host are essential for understanding. Up to now, PCR and then sequencing allowed huge progress in the description of life by allowing the tracking of genes and transcripts, and have revealed the vast genetic diversity and coding capacity of the microbiotas in human. Also, a lot of new epigenetic mechanisms have been revealed which makes more complex the understanding of really occurring events in the cells. To this purpose, functional studies of the microbial communities, the interactions among community members and with the host are essential and proteins remain in charge of structure and function in the cell. The reported discrepancy between expressed functions and functional potential suggests that caution should be used before drawing functional conclusions from metagenomics and metatranscriptomics data, further supporting metaproteomics as a fundamental approach to characterize the human metabolic functions and activities. LC-MS/MS


metaproteomics has benefited from advances in proteomics, PTMs and bioinformatics field, and is a promising approach to perform in-depth microbiome analysis as it simultaneously reveals both human and microbial changes. These improvements have led to a drastic increase in microbial protein identifications from 1000 proteins up to >10 000 proteins per microbiota. The limitations and challenges of metaproteomics include inadequate resolution of mass spectrometers to measure low-abundance proteins from complex microbial communities and the lack of an easy-to-use and high sensitivity metaproteomics computational workflow. However, the functional core proteins remain easily reachable and among the most impacted by external events. After bioinformatics analyses, important informations on occurring events can be described.

THE USE OF ‘LIVE’ BENEFICIAL LACTOBACILLI TO TREAT SKIN CONDITIONS

Abstract: YUN is a Belgian biotech company that succeeded in building an innovative and unique R&D platform and technology, allowing to keep bacteria alive in water-based environments. Use of live beneficial lactobacilli provides an antibiotics alternative by impacting the skin microbiome. Specifically for acne vulgaris YUN developed a formulation with microencapsulated live bacteria for the treatment of acne. Besides the impact on the skin microbiome and reduction of specific skin pathobionts linked with acne vulgaris, clinical symptoms of all patients with mild to moderate acne improved in our clinical trial. The improvement in clinical symptoms of acne is further underlined by a post-market follow up study. With our current data, YUN believes this will initiate a new era of skin therapeutics based on microbiome modulation, and provides a new therapeutic option to reduce antibiotic use for common skin conditions, like acne.

Cédric Bernarde is a biochemist. He made his PhD within an INSERM / Bordeaux University laboratory, on Helicobacter pyloriproteome and complexome. Then, he worked on bacterial secretion systems at the CEA Grenoble, and then at the Pasteur Institute in Paris. Next, he focused his researches on microbiotas. Particularly, he worked on the airway microbiota at the Brest University and finally on the intestinal microbiota within an INRA / Clermont Auvergne University team. Now, he is the Head of Bioinformatics and Biology at Phylogene SA, a company servicing in the omics field with a focus on microbiotas.

Cédric Bernarde | Phylogene SA

Ingmar Claes is the head of science (CSO) at YUN. Ingmar studied bioscience engineering at the University of Leuven, graduated in 2007 and obtained a PhD in cell and gene technology in 2011. He has an expertise in the field of probiotics, its molecular mechanisms and the topical application of these beneficial bacteria to restore the human microbiome. As bioengineer and microbiologist, he feels that it is important to translate this scientific research into daily products from which people at home and the community can benefit.

In the context of YUN, he is closely working together with the

Ingmar Claes | YUN NV


HANDLING A SKIN MICROBIOME PROJECT: SCIENTIFIC WATCH ASSOCIATED WITH BEST PRACTICS

Abstract: Study design for skin microbiome research is multifaceted and integral to all downstream steps. Many published studies examined the biases introduced by the skin sampling methods (1) (2) and sample storage (3), controls and contamination sources (4), sequencing biases (5), and possible quantitation (6). The complexity of skin microbiome studies is nicely summarized in the title of the review from (7) “Performing Skin Microbiome Research: A Method to the Madness“. Thus, standardization and validation of the protocols, bioinformatics pipelines and sequencing platforms are crucial and key parameters of a successful skin microbiome analysis.

During my talk, I will review the di�erent biases influencing skin Microbiome metagenomic studies, such as errors introduced during sample preparation, sequencing and analysis. I will also present an example of a global validation and harmonization approach (sequencing platforms and harmonization of the methods usedfor skin microbiome studies). In conclusion, a quality chart of skin microbiome projects will be proposed.

References:

1. Chng KR, et al. (2016) Whole metagenome profiling reveals skin microbiome-dependent susceptibility to atopic dermatitis flare. Nat Microbiol 1(9):16106.2. Grice EA, et al. (2008) A diversity profile of the human skin microbiota. Genome Res 18(7):1043-1050.3. Lauber CL, Zhou N, Gordon JI, Knight R, & Fierer N (2010) E�ect of storage conditions on the assessment of bacterial community structure in soil and human-associated samples. FEMS Microbiol Lett 307(1):80-86.4. Salter SJ, et al. (2014) Reagent and laboratory contamination can critically impact sequence-based microbiome analyses. BMC Biol 12.5. Meisel JS, et al. (2016) Skin microbiome surveys are strongly influenced by experimental design. J Invest Dermatol 136.6. Gao Z, Perez-Perez GI, Chen Y, & Blaser MJ (2010) Quantitation of major human cutaneous bacterial and fungal populations. J Clin Microbiol 48(10):3575-3581.7. Kong HH, et al. (2017) Performing Skin Microbiome Research: A Method to the Madness. J Invest Dermatol 137(3):561-568

Microbiology and Biotechnology of prof. Sarah Lebeer, the Lab of Pharmaceutical Technology and Biopharmacy of prof. Filip Kiekens, prof. dr. Julien Lambert (dermatology) and dr. Gilbert Donders (gynaecology, the University Hospital of Antwerp).

He has experience with leadership in multiple R&D projects and he held a managing role in YUN’s R&D projects of the Flemish Institute for the Promotion of Scientific and Technological Research in Industry (VLAIO). YUN’s current range of probiotic products and therapies were developed under his supervision.

After his “Maîtrise” in Biochemistry in Lebanon, Ahmad was selected by the International Scholarship program of the Ecole Normale Supérieure de Cachan to do my Masters in Microbiology and Biochemistry.

G. Ahmad Khodr | L’Oréal Research and Innovation


Dr. Tompkins completed his doctorate studies in Biochemistry at the Hospital for Sick Children in Toronto and his post-doctoral studies at the University of Guelph. As Research Director at Lallemand Health Solutions since 1999, his teams evaluate the safety and action of probiotics and their e�ect on immunity in human cell systems. His current focus is the role of the microbiota, and its associated resistome in gastrointestinal and neurological disorders. He collaborates with a number of external investigators to evaluate clinical opportunities for the use of microbes.

Thomas Tompkins | Lallemand Health Solutions

A COMPREHENSIVE REVIEW OF PHARMABIOTIC COMPOSITION IN THE MANAGEMENT OF IRRITABLE BOWEL SYNDROME IN CHINESE POPULATION

Abstract: Medilac-S (also known as IBacilluS+) is a pharmabiotic sold by Hanmi Pharmaceuticals in Asia, primarily China and Korea, where it is registered as a pharmaceutical. It is composed of two probiotic bacteria, Enterococcus faecium R0026 and Bacillus subtilis R0179. This product has been used in a number of applications including the management of symptoms of Irritable Bowel Syndrome (IBS). In a systematic review of the literature, we identified more than 100 publications describing the clinical intervention in patients with IBS with this composition in China, between 2000 and 2018. Patient inclusion was primarily based on the Rome criteria at the time. Nearly half the papers report on the diarrhea-type IBS, the remaining papers report outcomes in constipation-type IBS or global IBS. Positive outcomes, in terms of clinical e�cacy, were reported in most of the studies. The majority of the studies were performed in adults, but there were a few in teenagers and only two studies in children less than 12 years with a children’s formulation, Medilac-Vita. This presentation will examine the strength of the clinical evidence and make recommendations as to the appropriate dose, duration of treatment, age group and the realistic expectations of improvement for the various types and symptoms of IBS.

Following his Masters, he conducted a Ph.D. research program in Microbiology & Molecular Biology at the ENS Cachan, France focusing on the regulation of virulence of Enteropathogenic E. coliby H-NS family proteins: di�erential regulation of LEE operons by H-NS and Ler proteins in EPEC. He carried-out a post-Doc research program at the Pasteur Institute Paris, where he applied Transcriptomics and Next Generation Sequencing tools to decipher virulence mechanisms of Legionella pneumophila. Recently, he joined the International Microbiology department in L’Oréal Research and Innovation as a Research Scientist/Manager where he leads an Expertise Working Group that aims to build aMicrobiome evaluation toolbox in vitro and standardize/validate clinical/skin Microbiome metagenomic studies and data. He has published and presented his research work in well recognized international peer reviewed journals and congresses.


The Microbiome & Cancer

PRECISION THERAPY IN COLORECTAL CANCER: DEFINING THE ROLE OF THE GUT MICROBIOME

Abstract: Sporadic colorectal cancer (CRC) remains the second leading cause of cancer deaths in the UK and it is a disease whose aetiology is defined primarily by interactions between the host and his environment. The gut microbiota, which sits at the interface between the host’s physiology and exposome, is now recognised as a critical modulator in CRC aetiology. However, our understanding of this immensely complex, highly individualised and multi-faceted relationship is incomplete. We have demonstrated that gut microbiota composition is indicative of CRC disease stage and molecular phenotype, and we have defined anti-correlated community sub-types, that defines this association. Utilising liquid-chromatography mass spectrometry we have analysed how metabolomic changes in the developing tumour micro-environment provide insight into the microbial driver and passenger co-metabolic functions that in turn influence cancer phenotypes and treatment response. Moreover, we have mapped how the gut microbiome influences chemotherapeutic e�cacy and toxicity through the proposed TIMER framework. We now propose a novel model for future precision therapy in colorectal cancer and outline future opportunities for engineering the microbiome for improved patient outcomes.

James Kinross is a Senior Lecturer and conusultant colorectal surgen at Imperial College London. He was awarded his PhD in 2010 and has published extensively on the gut microbiome. His research group is studying how the gut microbiome initiates colorectal cancer and exploring how it may be engineered to improve patient outcomes from surgery and chemoradiotherapy.

James M. Kinross | Imperial College London

THE ROLE OF THE MICROBIOTA IN REGULATING BREAST CANCER

Abstract: Modulation of the gut microbiota has been shown to influence primary tumour growth across multiple diseases; however, despite breast cancer being so common in the Western World, there have been a paucity of studies addressing the role of the microbiome. We have shown that antibiotic induced dysbiosis accelerates primary tumour growth. However, to our surprise this appears to be largely driven by metabolic changes rather than immunological modulation. We have also shown that the microbiome plays a role in guiding and controlling metastatic breast cancer by influencing the metastatic niche. Overall, these findings have demonstrated a key role for the microbiome in breast cancer growth and progression. Our data suggests that antibiotic use in BC patients should be examined closely and re-evaluated to avoid comprising treatment e�cacy.

Stephen started his scientific career studying mammary gland biology at the University of California, Santa Cruz. He then conducted his postdoctoral work at the Massachusetts Institute of Technology, where he developed Selectin double- and

Stephen Robinson | Quadram Institute


METAGENOMICS FOR EVALUATION OF CHEMOTHERAPY IMPACT ON GUT MICROBIOME

Abstract: It is now well established that, besides genetic features, one major host determinant in human health is the gut microbiome. The French consortium IMODI (Innovative MODels Initiative) aims to develop predictive preclinical mouse models for new chemotherapeutic treatment discovery, to progress toward personalized medicine. In this consortium, Biofortis Mérieux NutriSciences issue is to better understand how the treatment can impact the gut microbiome of patients and of mouse models named Patient-Derived Tumor Xenograft (PDX). In this purpose, we coordinate a clinical study to recruit patients with four types of cancer (breast, ovary, liver and prostate) from french clinical centers. Stool samples from patients and PDX are collected and the gut microbiome composition is assessed by 16S rRNA gene sequencing before, during and after chemotherapy. The taxonomic classification of sequences is obtainedusing bioinformatics and statistics skills. Our work shows that some chemotherapeutic agents could have a specific e�ect on gut microbiome. Imodi stool and blood biobank is available for the scientific community. This study supports that the microbiome is an important additional parameter for evaluating the toxicity of pharmacological treatments. The evaluation of the microbiome in the development of new therapeutic agents is highly recommended.

triple-knockout mice and showed that P-selectin plays a dominant role in leukocyte tra�cking. In 2000, Stephen moved from to the UK to work with Professor Kairbaan Hodivala-Dilke at Barts Cancer Institute. In 2011 he became an independent group leader at the University of East Anglia (UEA) and in 2018 he became a Research Leader at the Quadram Institute with their major aims being to understand how microbiome-host interactions regulate health and disease, with major focuses on vascular development and homeostasis and cancer.

Dr. Françoise Le Vacon is an expert in Molecular Biology. In 1996, she founded Atlangene Applications, which is now integrated within Biofortis. Françoise obtained a Master’s degree in Cellular and Molecular Biology from the University of Rennes, France, in 1985. She then obtained a PhD from the Faculty of Sciences at the University of Nantes, France, having carried out work of key importance at the INSERM U211 laboratory under Prof. Francine Jotereau, on the subject of “Murin’s Lymphomas induced by Abelson Virus” in 1990. Following various assignments at the Hôpital Cochin and Institut Pasteur in Paris, and the Johns Hopkins Hospital and National Institute of Health in the U.S., she has held research and development managerial positions for the last 20 years. She has also worked as a researcher at the René Gauducheau Centre for Cancer Care in Nantes, and as a laboratory manager at the Grenoble Transfusion Center. She joined Silliker France in 2007 as the Molecular Biology Laboratory Manager in Food Quality and Safety, with expertise in food allergens. As

Françoise Le Vacon | Biofortis Mérieux NutriSciences


Exploring the Gut-Lung Axis

Microbiota Research Program Director at Biofortis, Françoise set up a platform for the study of bacterial ecosystems for nutrition and health. In 2011, Françoise received the French National Order of Merit for her contribution to science.

THE LUNG-GUT AXIS

Abstract: The Gut-Lung axis assumes that the microbiology of the gut influences respiratory disease via a number of poorly described mechanisms, while frequently failing to account for the local microbiology in the lungs. Here, I explore the axis, present evidence for a Lung-Gut axis and demonstrate how experimental approach can profoundly influence findings in microbiome studies.

Michael Cox trained in Marine Microbial Ecology, but for the last ten years has been applying these techniques to the respiratory microbiome. His interests are in chronic and acute respiratory diseases, in how microorganisms interact with their host environment and in how to do a proper experiment.

Michael Cox | Imperial College London

TITLE TO BE CONFIRMED

Abstract: Coming Soon.

BIO Coming Soon.

Philip Molyneaux | Royal Brompton Hospital


Developing Synthetically Engineered Pharmabiotics

UNCOVERING SPECIES AND STRAINS IN THE HUMAN MICROBIOME FOR PERSONALIZED INTERVENTIONS

Abstract: The full diversity of the body-wide human microbiome remains partially uncharacterized, especially at the level of single strains and in international populations. I will describe our recent findings in uncovering many hundreds of previously uncharacterized species, identifying condition-linked microbial strains, and detecting specific variants of bacterial enzymes connected with diseases such as colorectal cancer. I will discuss how these findings can potentially result in novel microbiome-driven or microbiome-mediated therapies.

Professor Nicola Segata is a computational biologist at the CIBIO Department at University of Trento (Italy). His lab employs experimental meta'omic tools and novel computational approaches to study the diversity of the microbiome across conditions and populations and its role in human diseases and infections. The projects in the lab bring together computer scientists, microbiologists, statisticians, and clinicians and are generally focused on profiling microbiomes with strain-level resolution and on the meta-analysis of very large sets of metagenomes with novel computational tools.

Nicola Segata | CIBIO, University of Trento

Travis Whitfill is the Chief Science O�cer of Azitra Inc. His background began in molecular biology and biochemistry after receiving scientific training at the MD Anderson Cancer Center and Duke University. He has co-founded several biotech and healthcare startups companies, including Azitra. Mr. Whitfill’s strong background in entrepreneurship and business was recently acknowledged when he was named one of Forbes’ 30 Under 30 in 2018. He serves as a partner at Bios Partners, a

Travis Whitfill | Azitra Inc.

BUGS AS DRUGS FOR THE SKIN: ENGINEERING THE SKIN MICROBIOME FOR NOVEL THERAPEUTICS

Abstract: The skin microbiome plays a fundamental role in human health, protecting against pathogens and antigens while bolstering cutaneous immunity. Imbalances in the skin microbiome (i.e. "dysbiosis") are highly associated with severity of skin disease, and research shows that improving the skin microbiome may be a promising approach to treating disease. Novel strategies have emerged to harness the skin microbiome to treat a plethora of diseases. Azitra is developing a platform to treat atopic dermatitis and other skin diseases using protein-secreting S. epidermidis. The team has engineered these strains to secrete various human proteins and is developing AZT-01 (filaggrin-secreting strain) for atopic dermatitis, AZT-02 (LEKTI-secreting strain), and AZT-04 (non-secreting strain) for chemotherapy-induced rashes..


healthcare-focused venture capital fund based in Texas, and as the Senior Analyst at Bios Research, which provides research services to institutional investors. He also is an Associate Research Scientist in the Department of Pediatrics at Yale University. Mr. Whitfill received degrees from Yale University (MPH) and Dallas Baptist University (BS in biology).

DEVELOPMENT OF A NOVEL MICROBIOME THERAPEUTICS USING ENGINEERED CLOSTRIDIUM

Abstract: CHAIN has built a proprietary live bio-therapeutics platform based on a single clostridium species, that supports multiple therapeutic products. CHAIN’s products address an unmet clinical need for safer, more e�ective and lower cost drugs. Our treatments for diseases associated with the human gut microbiome have the potential to transform healthcare across many chronic and debilitating diseases.

For example, CHAIN’s lead asset produces a potent anti-inflammatory for treating ulcerative colitis. CHAIN has demonstrated safety and e�cacy in vivo, scaled and de-risked manufacture, and is now rapidly progressing this product towards the clinic. CHAIN is also developing several other products in parallel with a focus on infection, inflammation and metabolic disorders such as type 2 diabetes.

Ben Bradley is Head of Partnerships & Licensing. Ben joined CHAIN Biotech in 2014, shortly after company inception and has played key role in the company’s development, helping to drive a strategic switch from classic Industrial Biotech into a company now developing and establishing a position within the microbiome-based therapeutics field. Ben has a background in molecular genetics and biotechnology and is part of the commercial team at CHAIN.

Ben Bradley | CHAIN Biotechnology Ltd.

MANIPULATION OF THE MICROBIOME BY TARGETED DELIVERY OF DNA PAYLOADS WITH PHAGE-DERIVED PARTICLES

Abstract: Coming Soon.

BIO Coming Soon.

Xavier Duportet | Eligo Biosciences

Navigating Regulatory Pathways for Your Microbiome Products


BUGS AS DRUGS AND THE DEVELOPMENT & MANUFACTURING PROCESS

Abstract: Quality plays a major role in the safety and e�cacy profile of LBPs. It is the responsibility of the LBP developer & manufacturer to ensure that the manufacturing process is adequate and that appropriate measures are put in place to safeguard the quality of the product.

Adrien Nivoliez is the Chief Executive O�cer at Biose industrie (Drug GMP for Live Biotheraputic Products). Prior to this position, he has worked for several years at Probionov and Lyocentre with quality, production and global R&D positions. Adrien has completed his studies at the Université Paris-Sud, his PhD in Medical Microbiology and bacteriology at the University Blaise Pascal and had also gained an MBA from the European Institute of Technology (EPITECH). A major part of his research has been focused on the manufacturing impact on Live Biotherapeutic Product potencies.

Adrien Nivoliez | Biose Industrie

PROBIOTICS AND THE CODEX INITIATIVE: HISTORY, CURRENT SITUATION AND FUTURE PERSPECTIVES

Abstract: An expert consultation group, called together by the Food and Agriculture Organization/World Health Organization (FAO/WHO) was held in 2001 on the “Evaluation of health and nutritional properties of probiotics in food, including powdered milk with live lactic acid bacteria”. The importance of probiotics to human and animal health was recognized and many aspects of the use of probiotic foods and their production were discussed. The working group intended to overcome a lack of international agreements on how to define and label probiotics and how to assess their safety and e�cacy, involving aspects of production, dose and target population. Eighteen years later there is still no consensus on many of these aspects. The ban of the word ‘probiotic’ in Europe, because of the definition proposed in 2001, is only one of the pending issues which requires a solution. The lack of harmonization in industry practice and legislation is confusing for regulators, industry, and even for the consumer. Many countries, led by Argentina, recognize the need and opportunity for development of a Codex Alimentarius guidance or standard to more clearly define the required characteristics of safe and e�cacious probiotics, and ensure the same level of quality and manufacturing requirements for all operators on the market. The Codex Alimentarius international food standards and guidelines contribute to the safety,

Dr. ir. Bart Degeest graduated in 1995 as an engineer in cell- and gene-biotechnology at the Vrije Universiteit Brussel (Belgium) and obtained in 2001 a PhD in Applied Biological Sciences at the same university with a research project on the composition and production of exopolysaccharides by Streptococcus thermophilus. After his PhD period Bart joined the company Yakult as Science

Bart Degeest | Yakult Europe

manager, taking care of research projects and scientific communication in the field of probiotics. He is now managing director of Yakult Belgium & France and very much involved in, besides the ongoing research, the activities of working groups and organisations as FEVIA, ILSI, IDF, IPA-EU, IPA-global and CODEX. The objective of these organisations is often related to a better definition of probiotics, their safety, their production process and, in a European context, the re-introduction of the term ‘probiotic’.


Magali Cordaillat-Simmons earned her PhD in 2005 at Université Descartes - Paris, France, in Cardiovascular Pharmacology. She then worked as a Research Associate at the University of Virginia, where she studied the influence of epigenetics on renin expression. Today, she is the Executive Director for Scientific & Regulatory A�airs at the Pharmabiotic Research Institute (PRI), located in France. The PRI is Europe’s first and only expert regulatory group dedicated to supporting its members in the development and market authorization of Microbiotic Medicinal Products (MMPs). As a non-profit, Pro-industry organization, the PRI is uniquely positioned to both be a trusted point of contact for the European regulatory authorities, as well as an agile and neutral partner for the Microbiome & Health industries. Dr Cordaillat-Simmonscurrently supervises the Association’s analysis of the European Pharmaceutical Legislation as well as the PRI’s unique regulatory intelligence strategy so that “Microbiotic Medicinal Products” may become a therapeutic reality for European patients.

Magali Cordaillat-Simmons | PRI

PUTTING INTO PERSPECTIVE THE INTERNATIONAL REGULATORY INITIATIVES IN FOOD & PHARMA

Abstract: Regarding the latest developments surrounding food and pharma regulations, it would seem important to clarify the di�erent initiatives and changes relative to living micro-organisms and their consequences for developers. On the pharma side, clarification of the expectations of drug authorities is of the utmost importance.In this presentation, we will address certain key points facing the industry today: medical device status and robust clinical design, among others…

ISO-IDF STANDARDIZATION OF FLOW CYTOMETRY METHODS FOR BENEFICIAL MICROBES

Abstract: Recent advances of research in microbial analytics have led to the development of next-generation flow cytometry protocols. These innovative approaches are particularly well suited for global and multiparametric analysis of probiotics; and they bring e�cient solutions to the numerous limitations of classical microbiology and other analytical methods.In this context, standardization of these new methods is a critical requirement for their harmonized implementation and application. In 2015, the International Standardization Organization (ISO) and the International Dairy Federation

LCDR Matthew Steele is a Team Leader in Regulatory Review Branch 1 in the Division of Vaccines and Related Products Applications (DVRPA), in the O�ce of Vaccines Research and Review (OVRR) at CBER. Additionally, he is a Lieutenant Commander in the United States Public Health Service Commissioned Corps. LCDR Steele has been with the FDA since 2009. During that time he has served as the primary reviewer for numerous pre-IND meetings, INDs, and BLAs. His primary focus has been live microbiome-based products and live biotherapeutic products. He serves as the primary POC for CBER for fecal microbiota

Matthew Steele | US FDA

LIVE MICROBIOME-BASED PRODUCTS USED TO PREVENT, TREAT, OR CURE DISEASES IN HUMANS: CLINICAL AND CMC CONSIDERATIONS

Abstract: The O�ce of Vaccines Research and Review (OVRR) in the Center for Biologics Evaluation and Research (CBER) is responsible for regulatory review of Investigational New Drug (IND) applications and Biologics License Applications (BLAs) for preventive vaccines and related biological products, including live microbiome – based products used to prevent, treat, or cure diseases in humans. Through this review process, OVRR ensures that the products are safe, pure and potent. During the IND review process, the amount and type of data changes as drug development progresses. My presentation will highlight the clinical and CMC expectations at each phase of development for microbiome-based products.

(IDF) published a standard on flow cytometry validated for the use in quantification of Lactic Acid Bacteria (LAB) and probiotic strains (ISO 19344:2015 | IDF 232). This first ISO/IDF standard includes three individual protocols, each one determining di�erent viability parameter. Equivalence between the three staining protocols has been demonstrated. ISO/IDF has recently initiated another flow cytometry standardization project aiming at specific quantification and viability assessment of multiple microbial species in LAB and probiotic products. This presentation will give an overview of the current situation regarding standardization of flow cytometry methods applied to beneficial microbes as well as future perspective.


Dr Pierre Burguière is a microbiologist with 15+ years of experience. He’s the founder and CEO of AMA Research Solutions, a biotech company servicing in Flow Cytometry to develop innovative solutions for the analysis of beneficial microbes for health (i.e. live biotherapeutics) and microbiotas. Dr Burguière is also Delegate for France on the Standing Committee for Analytical Methods for Dairy Microorganisms (SCAMDM) of the International Dairy Federation, member of the U.S. Pharmacopeial Convention Expert Panel on Probiotics, and member of the Board of Directors of the French Association for Cytometry.

Pierre Burguière | International Dairy Federation / AMA Research


transplantation. Prior to joining the Agency, LCDR Steele earned a B.S. in Biology at Salisbury University (Salisbury, MD) and a Ph.D. in Biochemistry and Molecular Biology at the Johns Hopkins Bloomberg School of Public Health (Baltimore, MD). He completed his Ph.D work in the labs of Dr. Cecile Pickart and Dr. Michael Matunis, and wrote his thesis on kinetics of deubiquitinating enzymes.

Biotech ShowcaseHARNESSING BIOFILMS TO ENHANCE BACTERIAL THERAPEUTICS

Abstract: Based in Indianapolis, IN and Boston, MA, Scioto Biosciences is a clinical stage microbiome company developing innovative therapies devoted to having a transformative impact on the delivery of live bacterial therapeutics (LBTs). Scioto was founded in 2017 as a partnership between Indiana business accelerator, Monon Bioventures (MBV) and The Research Institute at Nationwide Children's Hospital(RINCH). The Scioto Activated Bacterial Therapeutic (ABT) Platform has the potential to enhance e�cacy and potency wherever LBTs are used such as diabetes, neurological disorders, gastrointestinal health, alternatives to in-feed antibiotics (in livestock) and others. For more information, visit http://sciotobiosciences.com/.

Joe is Co-founder and CEO of Scioto Biosciences and Monon Bioventures. He brings over 15 years of experience in early stage asset development to the team. Joe was the Head of Startups at the Indiana University Research and Technology Corporation and was responsible for assisting Indiana University faculty, sta� and students form and run more than 25 startup companies. The success of the program inspired the formation of Monon Bioventures (MBV). MBV is a privately held company specializing in early stage venture creation and management.

Throughout his career, Joe has served in technology transfer at the Purdue Research Foundation managing a wide range of portfolios in the medical device, software, pharmaceutical, agriculture, and chemical disciplines. Previously, he worked as a Principle within the venture capital group IN-Vivo Ventures and served as Vice President of Research and Development for Kylin Therapeutics. Joe has a PhD in Pharmacology from Purdue University and an AB in Chemistry from Wabash College.

Joe Trebley | Scioto Biosciences

AUTOMATED METATRANSCRIPTOMIC TECHNOLOGY: DATA-DRIVEN PERSONALIZED NUTRITION AND MEDICINE


LEVERAGING THE THERAPEUTIC POWER OF MICROBIOMES WITH THE NANOFLEMING PLATFORM

Abstract: Untreatable bacterial infections, exacerbated by the rise of antibiotic resistance, are undoing the successes of modern medicine. We need new e�ective therapies. Microbial communities have evolved formidable strategies to fend o� invaders and competitors. At SpheroBiotics we have built a proprietary discovery platform to harnesses the unexplored therapeutic potential of microbial communities – the nanoFleming. Working at the nanoliter scale, we probe millions of microbial interactions in parallel to isolate “defender” strains that suppress pathogens. We cultivate and assay microbiota in a close approximation of their natural habitats. The nanoFleming has been successfully applied to screen engineered strains for production of antibiotics, and we are now expanding the technology to human and soil microbiomes. This will allow us to unlock a vast treasure trove of valuable novel antibiotic medications and live biotherapeutics.

Irene Wüthrich is a co-founder of SpheroBiotics, a start-up from ETH Zürich that leverages microbial communities as a source for novel antibiotics and live biotherapeutics to treat bacterial infections. Irene has a “diplôme d’ingénieur” and MSc in biotechnology from the Ecole Supérieure de Biotechnologie in Strasbourg, France, earned her PhD in molecular biology at the Whitehead Institute for Biomedical Research at MIT in Cambridge MA, USA, and is a synthetic biologist and Pioneer Fellow at ETH Zürich, Switzerland.

Irene Wüthrich | SpheroBiotics

ADVANCING PRECISION MEDICINE THROUGH MICROBIOME-BASED THERAPEUTICS



Dr. Nikole Kimes is Founder, President and Chief Scientific O�cer of Siolta Therapeutics, a clinical stage biotech company developing novel microbiome-focused strategies for the prevention and treatment of inflammatory diseases. As the lead executive driving Siolta’s early-stage development, Dr. Kimes heads a talented team of scientists, blending microbiology, immunology, and bioinformatics expertise to leverage microbiome data for the improvement of patient stratification and development of precision microbial therapeutics. Dr. Kimes has over a decade of experience in microbial ecology and is an inventor of Siolta’s technology, her research in Dr. Susan Lynch’s lab at UCSF provided the foundation for the company’s translational research program.

Nikole Kimes | Siolta Therapeutics

abstracts & speaker proﬁles - pharmabiotics event · 2019-02-14 · abstracts & speaker...

Documents