June 27-29 2017

Boston, MA

Day One
Wednesday June 28, 2017

Day Two
Thursday June 29, 2017

Registration & Networking

Chair’s Opening Remarks

Optimizing Drug Target Validation and Candidate Selection

Introduction and Purpose:


It’s clear that research in the microbiome is redefining our understanding of human health and disease. Using sophisticated science, the microbiome can provide alternative approaches to known targets, as well as elucidate new targets.

The purpose of this theme will be to demonstrate novel research that substantiates the relationship between microbial diversity, function and causation within human disease.

Mining the Microbiome for New Therapeutic Candidates for Chronic Diseases


  • Understanding how the microbiome produces many bioactive molecules that can communicate directly with host receptors/pathways and exert direct effects on the host
    • Elucidation of the mechanisms by which bacterial bioactive molecules influence host
    biology is the new frontier in microbiome drug discovery and development
    • Deconvoluting the biological mechanisms involved to accelerate new pharmaceutical
    discovery by mining the microbiome

Culturing The “Unculturable” to Harness the Therapeutic Potential of the Human Intestinal Microbiome

Control of the Appetite Through the Microbiome


  • Establishing a link between human appetite and the microbiome
  • Understanding how TargEDys intend to control appetite and body weight using probiotic methodology
  • Preparing for in human trials through analysis of preclinical results

Microbiome Tools and Trends for the Pharmaceutical Industry

  • Julia Cope Director of Scientific Operations, Diversigen


• Ideal study design in microbiome projects is key to delivering actionable results.
• Industry standards on quality are nascent and expanding
• Increased emphasis on providing analysis of the microbiome across disease states.

Morning Refreshments and Speed Networking

Predictive Preclinical Packages – Utilising In-Vitro Pharmacology, Animal Models and Bioinformatics for Microbiome Research

Introduction and Purpose:


Prior to initial Phase 1 studies, a preliminary characterization of a microbiome therapeutic has to be made using a
combination of in-vitro/in-silico assays and animal models .
The purpose of this theme will be to outline the most predictive preclinical strategies that address key regulatory and
safety considerations whilst guaranteeing translational and clinical success.

Microbial Adaptations of the Microbiome


• Delivering concepts and tools that target microbial integration in the microbiome
• Understanding how persistence probabilities in the microbiome can be learned from
longitudinal studies
• Using metatranscriptomics to show microbiome response to new microorganisms

How to Better Utilize Analytics for Advanced Microbiome Classification & Pathway Prediction


  • Evaluating best ways to analyse microbiome datasets to discover patterns in health and learning how to use scalable analysis to differentiate between healthy and diseased subjects
  • Understanding the possibility of finding non-invasive diagnostic tools in microbiome ecologies

Rethink the Preclinical Paradigm to Identify Promising Therapeutic Candidates


  • Development of new risk assessment matrix to select the best candidates
  • Screen of indication enables to identify the best therapeutic area
  • The choice of animals models for the preclinical regulatory experiments have to be based on their metabolic and microbiome specificities

Growth Dynamics Based Target Discovery


  • Using patterns of metagenomic sequencing read coverage for different microbial genome, we provide a quantitative measure of a species’ growth rate
  • We demonstrate both in vitro and in vivo, under varying growth conditions, and in complex bacterial communities correlations of growth rates with various phenotypes

Lunch & Networking

Early Stage Translational Development- Delivery Systems for Patients

Introduction and Purpose:


Pioneering microbiome drug developers must devise a suitable formulation and drug delivery system that ensures proper drug delivery parameters. Despite the broad definition, microbiome therapeutic companies are implementing a variety of delivery systems including: Consortia, Single bacterium, Synthetic Biology or Metabolite/Small Molecule.

The purpose of this theme will be to review drug delivery systems that are currently used in industry and how different methodologies effect stability, efficacy, safety and dose ranging for human trials.

Targeted Delivery of Cells and Proteins to the Lower Gastrointestinal Tract for Local Therapeutic Action: Opportunities for the Development of Microbiome-Sparing Antimicrobials


  • Two critical formulation challenges limit the development of microbiome-based therapeutics: Effective drug delivery and scalability
  • Symbiotic Health has developed a suite of scalable formulation technologies for the delivery of recombinant proteins and bacteria to the lower GI tract for local therapeutic action
  • Recombinant phage lysin enzymes can be engineered to be ultra-narrow, microbiomesparing antimicrobials, with the potential to address critical public health challenges

Designing Microbial Therapeutics with a Human-First Discovery Platform


  • How to study microbial therapy engraftment
  • How to leverage adaptive trial design and FMT drug heterogeneity
  • Engineering and validating capsule-based delivery of microbial therapies

The Essential Ingredients for Decoding the Microbiome with Metabolomics

  • Kirk Beebe Director of Application Science, Metabolon


• Improving mechanistic understanding to enhance practical use of information
• An illustration to show how metabolites broker important microbe-host interactions
• Showing metabolomics as a tool to enrich insights into interaction associations

Drugs from bugs – How metagenomic dataset delivers druggable targets


  • Microbiome as a source of bioactives and novel targets
  • Quantitative and functional metagenomics
  • Gut-restricted therapeutics

Oral Delivery of Live Bio-Therapeutics: Drug Product Development for FIM Studies


• Key CMC considerations for the development of an LBT product.
• Critical factors affecting timelines when outsourcing GMP manufacturing activities of
LBT drug products.
• Incorporating ‘Quality’ (systems and regulations) into early phase dosage form design of LBT products

Afternoon Refreshments & Networking

Clinical Trial Design, Data and Patient Recruitment

Introduction and Purpose:


Despite the overwhelming importance of gathering effective clinical data to support microbiome therapies, there are still huge questions surrounding best practice in designing and monitoring successful clinical trials.

The purpose of this theme will be to determine the requirements necessary to develop safe and effective clinical trials.

The Next Generation Microbiome Clinical Trial


  • Understand the study framework for assessing the microbiome
  • Highlight the value of thoughtful translational endpoints in microbiome trials
  • Explore novel design elements for clinical trials tailored to the microbiome space

Clinical Trials with Microbiome Therapeutics: Rethinking Medical Therapy


  • The promise of microbiome therapies in medicine
  • Designing Clinical Trials for Microbiome Therapies
  • Therapeutic landscape and microbiome therapies

Developing a Successful Clinical Trial & Manufacturing Program for Effective Treatment against Lactose Intolerance


  • Defining clinical endpoints is a collaborative process that must balance regulatory, scientific and commercial factors
  • Managing a clinical and selecting a trial provider is dependent on the development stage
  • Think long-term when evaluating manufacturing partners and choosing between in-house or outsourced manufacturing

Human Microbiota: Proof of Concept to Production


  • General overview of live microbial production processes
  • Step by step process description with emphasis on process development
  • Considerations prior/ during tech transfer focusing on specific requirements for pharma (GMP) production

Realizing the Promise of the Microbiome: Using Rigorous Pharmaceutical Principles of Drug Development


  • Understanding a best-in-class therapy for treatment of rCDI and proof of principle for platform
  • Understanding how efficacy, regulatory, IP and patient preference components are fundamental to outlining a robust clinical program

Targeting Specific Microbiota to Influence Therapeutic Outcomes for Irritable Bowel Syndrome with Constipation (IBS-C)

  • Vince Wacher Product Development Consultant, Synthetic Biologics


  • Clinical overview of SYN-010 modified-release lovastatin lactone to inhibit methane production by intestinal M. smithii

Chairman’s Closing Remarks

End of Day 1