Program

8:00 am Registration & Breakfast


9:00 am Welcome Address

His Excellency Gérard Araud, Ambassador of France to the United States


9:15 am Panorama of Precision Medicine in Massachusetts

Travis A. McCready, President & CEO, MLSC

9:30 am Carnot Institutes: Research & Innovation for Industry and RTOs

Pascal Deschaseaux, VP Ai Carnot, General Manager, CALYM

  1. Read More: Abstract

    Carnot Institutes are a major French State-funded multi-disciplinary research network, building economic development through technologies and innovation. A Carnot Institute is a leading research organization which places partnering research, i.e. research led with and for companies, at the heart of its strategy. The 34 Carnot institutes represent together 15% of the French public laboratory workforce (27,000 research professionals) and more than 55% of France’s public research directly funded by the industry. The Carnot institutes collaborate with other Research and Technological Organizations internationally. The GLOBAL CARE Initiative, a co-organizer of the FAID 2016, is the consortium of the 5 human health-centered Carnot Institutes. It comprises the Carnot Institutes of Institut Pasteur (infectious diseases), of Institut Curie (cancers) and of ICM (CNS diseases), Voir & Entendre (vision and audition diseases and rehabilitation) and CALYM (lymphoma).


Session I "Big Data in Clinical Trials: From Integrated Patients Profile to Complex Biomarkers”

9:45 am A Multidimensional Contribution to Personalized Medicine in Lymphoma

Gilles Salles, Chairman, CALYM

  1. Read More: Abstract

    Lymphoma represents a heterogeneous group of malignant diseases originating from blood cells called lymphocytes. Although about half of lymphoma patients can be cured, there is a considerable variability in the success rate of therapy. Prognostic indexes developed in the 90’s were based on several clinical parameters and validated on cohorts of thousands of patients, thanks to the international collaboration of lymphoma experts. In the last 15 years, new biological tools and large scale genomic approaches allowed deciphering the intrinsic biological diversity of lymphoma tumors. Many biomarkers were identified and new stratifications models derived using a combination of those with clinical parameters emerged. Longitudinal follow-up of blood derived biomarkers were also able to predict the efficacy of therapies applied to patients. The inherited host genetic profile was also found to influence patients’ outcome. Meanwhile, the efficacy of therapy could be monitored with new imaging techniques. The integration of all these data represents today a real challenge addressed through different research programs conducted by LYSA, The Lymphoma Study Association. They will contribute to the development of personalized medicine and may help to design in silico models aiming to predict the clinical efficacy of a given drug, hence contributing to a faster drug development.

Michel Meignan, Professor of Nuclear Medicine, Henri Mondor University Hospital

  1. Read More: Abstract

    The imaging activities of the LYSA started in 2007 when we launched with the EORTC a cooperative trial based on the results of a centralized review of PET/CT performed under treatment to manage therapy. We built a system of online centralized review using Workstations settled in the main LYSA centers linked through a virtual private network. This allowed the results of a centralized review performed by 6 experts to be obtained within 72 hours. Imaging performed under treatment (interim PET) being more and more used to guide therapy in our subsequent trials and in many trials worldwide we moved towards a web based solution which allows each reviewer to report the images on its personal computer without uploading them. A data base was progressively acquired including now more than 7000 examinations of patients with various subtypes of lymphoma obtained before and after 2 or 4 cycles of treatment. From these data we were able to use imaging as a biomarker to predict outcome and to define new therapeutic strategy. We have defined new criteria of PET reporting (Deauville criteria) and new quantitative parameters of the response such as the ΔSUV. We now develop quantitative index from PET performed before therapy to evaluate the total metabolically active tumor burden. The idea is to stratify patients in different risk categories to tailor therapy according to the risk and to adjust the dosage of the drugs to the mass of the tumor. Encouraging results has been obtained from our group in different types of lymphoma. We have shown that the stratification given by the tumor volume can be refined with clinical or molecular data. The challenge is now to link imaging clinical and biological data in our data base to define new prognostic models and to optimize the new trials.

Fabrice Jardin, Director INSERM U918 Research Team, Centre Henri Becquerel

  1. Read More: Abstract

    Lymphomas constitute a highly heterogeneous group of hematological malignancies that has been partially deciphered in the last past decades by new molecular biology tools, including gene expression profiles (GEP) and more recently next generation sequencing (NGS) . In most of cases, the treatment is based on the combination of chemotherapy and immunotherapy (namely the worldwide standard “R-CHOP” regimen) that cures approximately 60% of the patients. In this context, LYSA (The Lymphoma Study Association) currently promotes several biological tests with the aim of tailoring therapies and favoring the emergence of the “post R-CHOP” era. For this purpose, new molecular tools that can be widely used in routine or to stratify patients enrolled in clinical triasl have been developed by LYSA. This includes the first NGS study of a large cohort of diffuse large B-cell lymphoma (the main lymphoma subtype) patients using a targeted gene panel (lymphopanel) and benchtop sequencers, focusing on genes identified as important for lymphomagenesis or whose potential has been pinpointed in Whole Exome Sequencing studies. Particular attention has been paid to the inclusion of actionable targets, highlighting potential candidate patients for novel personalized therapies. The “lymphopanel” has been also used to detect actionable mutations in cell-free circulating DNA in the setting of liquid biopsy. In addition to recurrent mutations detected by targeted NGS, GEP molecular tests that are likely to represent theranostic biomarkers in a highly competitive academic and non-academic context, have been developed by the LYSA group.

Peter Ho, CMO, Epizyme Inc

  1. Read More: Abstract

    The histone methyl transferase enhancer of zeste-homolog 2 (EZH2) is the catalytic subunit of the polycomb repressive complex 2 (PRC2) and responsible for methylation of lysine 27 of histone H3 (H3K27), which results in chromatin remodeling and repressed transcription when trimethylated (H3K27me3). Aberrant EZH2 activity has been implicated as an oncogenic driver in non-Hodgkin lymphoma (NHL). The SWI/SNF complex also remodels chromatin, activates transcription and acts in opposition to PRC2. Oncogenesis from mutation and/or loss of the SWI/SNF subunit INI1 in cancers such as malignant rhabdoid tumor (MRT) is sensitive to EZH2 inhibition. Tazemetostat is a potent, selective small molecule inhibitor of EZH2 that is in phase 2 clinical development in patients (pts) with NHL and advanced solid tumors (ST) that are INI1- or SMARCA4-negative. Epizyme has collaborated with LYSA on a phase 1 open-label first-in-human study to evaluate the safety, tolerability, and preliminary efficacy of tazemetostat administered orally as a monotherapy twice a day (BID). Eligible pts had either a relapsed/refractory B-cell NHL or ST. As of 7-Nov. 2015, 58 patients (pts) were enrolled to this trial. The Recommended phase 2 dose was determined to be 800 mg BID. Objective responses (CRs, PRs) were observed in pts with diffuse large B-cell lymphoma, follicular lymphoma, marginal zone lymphoma, MRT, epithelioid sarcoma and MRT of ovary. Tazemetostat has demonstrated a safety profile favorable for chronic dosing and objective responses in pts with relapsed or refractory B-cell NHL including DLBCL, FL and MZL and in subjects with advanced STs consisting of MRT, ES, and MRT of ovary. Phase 2 trials in B-cell NHL and INI1- or SMARCA4-negative tumors are enrolling.


10:25 amCoffee Break & Networking


10:55 amImaging as Analysis Tool and Biomarker

Pierre Tervé, VP Technic & Scientific Advisor, Keosys

  1. Read More: Abstract

    Keosys was founded in 2001 and quickly focused on PET imaging analysis devices. Keosys then extended its national exchange network and built the first Pan-European “near real-time” centralized review system for PET/CT imaging in 2007 with the Lymphoma Study Association (LYSA) for an EORTC trial.  As an addition to imaging analysis products this led Keosys to design the IMAGYS web platform as a CTIMS (Clinical Trial Imaging Management System). This platform typically helps to handle two challenging issues of any international multi-centric imaging trial: imaging de-identification and quality check at investigator sites and consistent imaging biomarkers analysis. Specifically, for PET imaging, acquisition protocol enforcement and proper de-identification are of utmost importance in order to get usable SUV values. Patient weight and uptake time are typically two crucial parameters. It is also crucial to use validated tools to get proper information from biomarkers like ΔSUV or MRI apparent diffusion coefficient (ADC). But collecting a high volume of imaging only data is not sufficient to find new imaging biomarkers and realize value from imaging “big data”. Like all other small data, aggregation with other data, for example biobanks or clinical information, is needed to extract data and furthermore extract knowledge. Through a network of supporting partners at imaging sites and data analysis & modeling experts, Keosys’ VICTORIA imaging big data project aims at getting knowledge from data based on an ethical model.

11:05 amKeynote Address - Becoming GE Digital

Jeff Caron, Chief Software Architect – Software Technology Office, GE Healthcare

  1. Read More: Abstract

    GE is becoming GE Digital, and the transformation across industries has begun.  The GE HealthCare presentation will feature a scan of technology strategies that GE is highlighting as the Industrial Internet for healthcare.  This will include a discussion of the “digital twin” predictive analytics and asset performance solutions, as well as a look forward into Precision Medicine.  GE and GE Healthcare are investing significantly in cloud based technologies, and this presentation will outline that strategy.

11:25 amA Virtual Laboratory for Image-based Neuroscience Discovery: Collaborative Research and Science Education in the Era of “Big Data”

Charles R.G. Guttmann, Director, Center for Neurological Imaging, Brigham & Women's Hospital, Associate Professor of Radiology, Harvard Medical School

  1. Read More: Abstract

    Digital medical imaging has become ubiquitous throughout the world. Every day, millions of patients are imaged for many clinical reasons, using modalities such as magnetic resonance imaging (MRI), computed tomography (CT), positron emission tomography (PET), digital mammography and digital X-ray systems. While automated image analysis tools continue to be developed, extracting quantitative information from complex biomedical images often remains a labor-intensive task for human experts. So while our enormous and growing archives of biomedical image data play roles in the care of individual patients, this resource has been barely touched for more ambitious research and data-mining purposes. We will present a web-based “virtual laboratory” that integrates project-specific data federation, user-friendly design and execution of experiments, as well as educational modules to train and certify human experts. SPINE (Structured Planning and Implementation of New Explorations) aims to accelerate research by integrating image-derived metrics with other biomarkers, intimately meshing research and education, and fostering the development of more effective and efficient image analysis approaches.

11:35 pmPanel I - Big Data & Imaging

Chair: Charles R.G. Guttmann, Director, Center for Neurological Imaging, Brigham & Women's Hospital, Associate Professor of Radiology, Harvard Medical School

Jeff Caron, Chief Software Architect – Software Technology Office, GE Healthcare

Pierre Tervé, VP Technic & Scientific Advisor, Keosys

Michel Meignan, Professor of Nuclear Medicine, Henri Mondor University Hospital


12:15 pmPitch Session

Quick-fire start-up pitches

Moderator: Brittany McDonough, Senior Manager of Business Development at MOITI (Massachusetts Office of International Trade and Investment)


12:45 pmLunch & Networking


1:45 pm Poster Session

Researchers showcase posters


2:15 pm Big Data Analytics for Acute Patient Care

Hans-Aloys WischmannHead of Philips Research North America

  1. Read More: Abstract

    A compilation of clinical data from tens of thousands of patients in intensive care units represents a unique treasure trove for developing and validating algorithms that will predict patient deterioration long before the vital parameters document an event. As a concrete example, an indicator for hemodynamic instability will be presented – including the partnership between Philips, MIT, and BIDMC to compile the database, the combination of machine learning and physiological modeling to achieve accurate predictions for each individual patient, the retrospective validation, the questions for the clinical trial with Barnes Jewish Hospital, and the benefits of sharing the data with the research community. Rounded off by an outlook on the ongoing work to extend this concept to the Emergency Department.

2:35 pm Next Generation of Clinical Trials in Oncology

Christophe Le Tourneau, Head of Early Trials, Institut Curie

  1. Read More: Abstract

    In oncology, genomics has practically entered the clinic with the emergence of molecularly targeted agents (MTAs). Some of these drugs have been approved with a companion diagnostic based on a specific genomic molecular alteration. However, MTAs have followed the same clinical development as chemotherapeutic agents and have been developed in selected tumor types. The occurrence of the same molecular alteration across various tumor types is well described. The latter raises the question of whether treatment decision in the future should be entirely based on molecular biology, independently of tumor location and histology. This approach can today be addressed in clinical trials, since major advances in high throughput technologies now make it possible to depict most druggable molecular alterations for an affordable cost in a time that is compatible with clinical practice. Studies using high throughput technologies have been initiated with the aim of personalizing therapy in oncology. I will present the different types of next generation of clinical trials in oncology.

Bridging Basic and Translational Research in Tumor Immunology for the Benefit of Cancer Patients

Emanuela Romano, Head of Immunotherapy Unit, Institut Curie

  1. Read More: Abstract

    Immunotherapy represents one of the most significant therapeutic advances in cancer in the past 10 years; in particular, immune checkpoint blockade arguably represents a milestone in modern medicine and will likely become a major treatment option for cancer patients, with increased long-term efficacy at reduced toxicity. Other types of immunotherapies, including adoptive cell therapy, bi-specific antibodies, and vaccines are being tested in early-phase clinical trials in solid tumors. Yet, immunotherapy only works well in a fraction of treated patients. The goal of the Immunotherapy Unit of the Institut Curie is to deliver rational therapeutic approaches to increase clinical benefit by bridging knowledge and resources from the Clinical Investigation Unit, the biobank, and the state-of-the-art technological platforms in immune-monitoring, mouse modeling, experimental pathology, and genetics. I will discuss how the integration of clinical and laboratory data could be integrated to inform the design of rational immunotherapies.

3:05 pm Keynote Address

Don Pettini, Sr. Director, Healthcare Product Strategy, Health Sciences Global Business Unit, Oracle

3:25 pm Squeezing Water from a Stone: Urine as a Biospecimen for RNA-based Biomarker Discovery

Jill A. Macoska, Director, Center for Personalized Cancer Therapy, University of Massachusetts, Boston

  1. Read More: Abstract

    Patient stratification and the choice of efficacious therapeutic strategies require the use of biomarkers that can distinguish between disease pathologies. This requires the acquisition of biospecimens from the patient, preferably in a non-invasive manner, and the identification and validation of biomarkers from cases and controls. In this talk, I will present progress on our studies to identify and validate RNA-based biomarkers from non-invasively collected human urine. These studies are aimed at distinguishing between proliferative, smooth muscle dysfunction, and fibrosis as pathobiologies contributing to lower urinary tract dysfunction (LUTD) in aging men. The identification of biomarkers that can distinguish between these pathobiologies will permit patient and therapeutic stratification to more effectively treat LUTD.

3:35 pmPanel II - Precision Medicine & Patient Interests

Chair: Jill A. Macoska, Director, Center for Personalized Cancer Therapy, University of Massachusetts, Boston

Michele K. Russell-Einhorn, Senior Director, Office for Human Research Studies, Dana-Farber Cancer Institute

Don Pettini, Sr. Director, Healthcare Product Strategy, Health Sciences Global Business Unit, Oracle

Christophe Le Tourneau, Head of Early Trials, Institut Curie


4:15 pm Coffee Break & Networking


4:45 pm Global Network Surveillance of Antimicrobial Resistance

Arnaud Fontanet, Director, Centre for Global Health, Institut Pasteur

  1. Read More: Abstract

    Context: While global antibiotic consumption continues to rise, the selection of increasingly resistant strains of bacteria and other pathogens continues to evolve. The problem of antimicrobial resistance is only exacerbated by a stall in antimicrobial innovation and discovery. Unaddressed, antimicrobial resistance has the potential to reverse over 70 years of medical advancement, with large economic and social costs. Project: the Institut Pasteur is launching a Global Network for Surveillance of Antimicrobial Resistance that will draw on the existing regional surveillance activities of the Institut Pasteur International Network, particularly in areas with the largest burden of infectious diseases and potential for antimicrobial resistance development. This project will establish a global subnetwork of resource hubs that will collect microbiological samples with relevant epidemiological and clinical data, determine antimicrobial resistance profiles through phenotypic and genotypic analyses, store samples in secured biobanks, and share sequencing and accompanying data through a high performance computing facility for joint analyses on pathogens of public health concern: bacteria, HIV, P. falciparum and M. tuberculosis. Expected outcome: The use of the latest ‘-omnic’ technology and pathogen diagnostics will provide a comprehensive, quantitative estimate of the emergence and spread of antimicrobial resistance around the world in order to inform and improve future public health measures.


5:00 pm Closing Remarks


8:00 am Registration & Breakfast


Session II "From Integrated Business Models to New Funding Strategies for Early Stage R&D”

8:30 am Keynote Address - The Institut Pasteur, a New Innovation Model

Christian Bréchot, President, Institut Pasteur

  1. Read More: Abstract

    The Institut Pasteur, an international research institute (33 Institutes in 26 countries) based in France, is characterized by an original financing structure, mixing research contracts and agreements, industrial royalties and R&D contracts with government contributions, public gifts, donations and revenues from assets. To ensure its sustainability, the Institut Pasteur is establishing a new innovation model and replacing the Institut Pasteur international network (IPIN) at the heart of our model, with the reinforcement of ties with the Institut Pasteur international network through incentive programs.

    The changes occurring in research funding have been addressed through an ambitious program to recruit talented scientists with attractive packages, nurturing of individual career tracks and a stronger administrative and career support to our researchers, original incentive programs and strong partnerships with industrials, worldwide. The implementation of this innovation model has been anchored on the creation of a Department of Development, the reinforcement and wider opening to the European and international environment of the Research Applications and Industrial Partnerships Department.

    In addition to a curiosity-driven strategy, the Institut Pasteur has created major incentive programs, called “Grands programmes fédérateurs”, which unite several laboratories of the Institut Pasteur in Paris and in the Institut Pasteur international network and foster long-term partnerships with industrial partners. The Institut Pasteur proposes its academic and industrials partners a modular “portfolio” of scientific offerings from the Institut Pasteur, consistent with the strategy of each partner. The creation of a dedicated taskforce on Ebola in 2014, gathering the Institut Pasteur and several institutes from the Institut Pasteur international network and leading to high-level scientific papers and to several diagnostic and therapeutic innovations, is a strong example of this new innovation model. These actions are also connected with our international fundraising efforts and the creation or reinforcement of scientific Foundations in the United States, in Switzerland and in Asia.

8:50 am Short Allocution

Robert G. Urban, Head of Johnson & Johnson Innovation Center, Boston

9:05 am Harrington Project

Jonathan Stamler, Director, Harrington Discovery Institute

  1. Read More: Abstract

    The Harrington Project for Discovery & Development is a $250 million US and UK initiative to accelerate the development of medical breakthroughs by physician-scientists into medicines that benefit patients. It is a unique model that aligns, through mission and structure, non-profit and for-profit resources into a new system for drug development. The Harrington Project addresses a set of major challenges in medicine that have created a development gap for promising discoveries and contributed to a long-term decline in new medicine approvals. Launched in 2012, The Harrington Project has already demonstrated its innovative model from the sourcing and development of breakthrough technologies to establishing strategic partnerships with disease foundations and pharmaceutical companies, all with a focus of advancing new medicines for patient benefit.

9:20 am From Academe to Startup: The Journey of an Entrepreneur at UCSF

Stephanie Marrus, Director, Entrepreneurship Center, University of California, San Francisco (UCSF)

  1. Read More: Abstract

    Entrepreneurship is thriving in academe these days. In this talk, we will describe the ecosystem that we have created at UCSF to form new ventures, some impactful ways of training scientists/technologists who lack a business background and the results to date in this effort to create a startup culture.

9:35 am Panel III - New Business Models of Innovation

Chair: Johannes Fruehauf, Founder & President, LabCentral

Robert G. Urban, Head of Johnson & Johnson Innovation Center, Boston

Stephanie Marrus, Director, Entrepreneurship Center, University of California, San Francisco (UCSF)

Jonathan Stamler, Director, Harrington Discovery Institute

Christian Bréchot, President, Institut Pasteur


10:15 amCoffee break & Networking


10:35 amSuccess Story: A Relevant Model for Academia-Industry R&D Partnerships

Gilles Salles, Chairman, CALYM

  1. Read More: Abstract

    A Relevant Model for Academia-Industry R&D Partnerships CALYM, one of the 34 France’s Carnot institutes, is the consortium for the acceleration of innovation and its transfer in the lymphoma field. It brings together 13 research entities (the LYSA and LYSARC non-profit research organizations and 11 public high level research teams) specializing in lymphoma, most frequent blood cancer. Thanks to a unique R&D offering, CALYM elaborates and runs many preclinical and clinical programs in collaboration with pharma, biotech, imaging and in vitro diagnostics companies to bring answers to unmet medical needs in lymphoma, from the identification of new cellular targets to international phase III registration trials. CALYM’s own research programs are organized through four pillars: (i) validation of new lymphoma biological targets and in vitro/in vivo models (ii) identification, validation, protection and out-licensing of lymphoma biomarkers (iii) acceleration of translational research through the identification of early pharmacodynamic signals of activity (iv) acceleration of development, registration, and commercialization of drug candidates through the optimization of tools, processes and platforms related to clinical research. The consortium has also developed a unique collection of cryopreserved living cells for the screening of new lymphoma therapies. Overall, CALYM offers the industry an opportunity of one-stop-shop access to a wide range of expertise, tools and platforms fort their research and development strategies in lymphoma.

Kenichi Takeshita, VP, Clinical R&D, Celgene Corporation

  1. Read More: Abstract

    A Relevant Model for Academia-Industry R&D Partnerships Developing new therapies for cancer patients requires strong collaboration between academia and industry. The fundamental basis for drug development starts with discoveries based on disease pathogenesis, typically made in academia. Industry partner brings its expertise in taking new experimental therapeutic agents to the clinical trial stage. Successful development of drugs to registration and approval requires continued industry-academia collaboration with each party bringing expertise to the collaboration. Celgene and LYSA / CALYM collaborations have resulted in accomplishments in many aspects of lymphoma, from translational research projects to clinical trials and surrogate endpoints.

10:55 amSuccess Story: Biological Integrated Approaches for Drug Development

Sergio Roman-Roman, Head of Translational Research, Institut Curie

Keith A. Hoffmaster, Director, Global Program Management, Translational Clinical Oncology, Novartis Institutes for Biomedical Research

  1. Read More: Abstract

    Robust preclinical data are mandatory to optimize early clinical trials and reduce the dramatic attrition rate in late clinical protocols in the field of Oncology. Institut Curie (IC) has developed a recognized know-how in preclinical oncology taking advantage of (i) the access to human samples of our Hospitals, (ii) the multidisciplinary skills of the Research Center and our clinicians, (iii) the access to state-of-the-art technological platforms, (iv) the expertise in cancer animal models and pharmacology, and (v) the capacity to identify biomarkers for patient stratification or resistance to therapies. The Institut Curie has implemented years ago a Translational Research Department gathering platforms, expertise and disease-devoted teams with the objective of helping scientists from both Academia and Industry to ensure the continuum cancer research/patient care at different steps. Very-well recognized by the quality of its basic research, IC has devoted a big effort in the last years to incentivize translational research and promote clinical research, and more specifically early clinical trials. We would like to illustrate the way IC interacts with industrial partners with a successful research program established with Novartis on a rare disease, uveal melanoma. This project has allowed in vitro and in vivo proof of concept studies extremely useful for setting up an international early clinical trial which will be coordinated by IC. Furthermore, promising drug combinations have been identified which will pave the way for new therapeutic strategies in this dismal disease.


Session III "Rare Diseases: Models and Opportunities”

  1. Read More: Abstract

    More than six thousand different types of rare diseases and disorders have been described, with more being discovered every day. In recent years, the interest for rare diseases has grown throughout the entire drug development community, fueled by the appetite for shorter clinical development timelines, supportive IP regulation and favorable regulatory environment enjoyed in the field : As a result, in 2014, the Food and Drug Administration approved a record 17 medicines in the field, while nearly 500 others are in development. Yet there is more to add to this excitement: Drugs for rare diseases often benefit from the strong financial involvement of patient-advocacy groups as well as from extensive registries of patients favoring the fast recruitment for drug studies, an area in which France has long been staying forefront. Last and not least, these drugs have substantial potential to become symptomatic treatments in other, more frequent diseases. This session will focus on the field of CNS and ophthalmic disorders, where some well-chosen rare diseases display a phenotypic richness such that they offer a unique opportunity to develop “1st in man” protocols able to deeply inform the design of future POC trials in other indications. Using the examples of Tourette syndrome, neuro-metabolic disorders and several others, panelists will discuss why and how the gate of rare CNS diseases can open large avenues of development for biotech and pharma groups.

11:15 amShort Allocutions & Panel IV - Rare Diseases: Models and Opportunities

Yves Agid, Founder & Scientific Advisor, ICM

José-Alain Sahel, Founder & Director, Institut de la Vision

Fanny Mochel, Head of Metabolic Research Group, ICM

Michel Vellard, VP of Research, Ultragenyx

Gerry Cox, VP Clinical Development Rare Diseases Group, Genzyme, a Sanofi Company


12:45 pmPitch Session

Quick-fire start-up pitches

Moderator: Brittany McDonough, Senior Manager of Business Development at MOITI Massachusetts Office of International Trade and Investment


1:00 pmLunch & Networking


2:30 pm Keynote Address - Moderna: Pioneering Clinical Messenger RNA Therapeutics

Stéphane Bancel , CEO, Moderna Therapeutics


3:00 pm Coffee break & Networking


Session IV "Transforming Clinical Research: New End-Points and Regulatory Renewal”

  1. Read More: Abstract

    Progress in understanding retinal biology in health and disease moved first therapies to cure blindness from bench to bedside. Spectacular technological innovations allowed hopes for successful partial vision restoration in blind people through gene and cell therapies and retinal prosthesis. The development of these emergent treatments typically involves evaluation of their efficacy in clinical trials. Assessment of the clinical benefit of these new therapies however remains challenging, particularly in the view of lack of standard therapies for blindness that could be used for comparison. Accordingly, new clinical trial endpoints and regulatory approaches for approval of these new drugs, biologics and devices are needed. Ideally, in addition to the efficacy endpoints (eg visual acuity), quantitative measurements of quality-of-life and other socioeconomic parameters should be included in the battery of clinically meaningful endpoints. Standardized tests for daily living task performance, orientation and mobility in lighted and dark environments can detect efficacy of the treatment in sensitive and reliable manner. Breakthrough high-resolution non-invasive imaging could not only serve for selection of patients for emerging therapies trials but can also be used to optime the ratio between potential hazards and expected functional benefits of the treatment.

3:30 pm Short Allocutions & Panel V - Transforming Clinical Research: New End-Points and Regulatory Renewal

José-Alain Sahel, Founder & Director, Institut de la Vision

Yves Agid, Founder & Scientific Advisor, ICM

Malvina B. Eydelman, Director, Division of Ophtalmic and Ear, Nose and Throat Devices, FDA

Newton Howard, Chairman, Brain Sciences Foundation

Daniel Chung, Medical Affair Ophtalmic Lead, Spark Therapeutics

  1. Read More: Abstract

    Gene therapy trials for inherited retinal diseases (IRD) have proliferated due to increased identification of IRD genes, and the improved ability to deliver gene-based therapeutics directly to target cells. Traditional endpoints such as visual acuity and visual fields may not appropriately capture improvements in functional vision, necessitating the development of new clinically meaningful endpoints, such as multi-luminance mobility testing.


5:00 pm Poster Awards & Concluding Address

Nahid Bhadelia, Infectious Diseases Physician, Boston University School of Medicine, Director of Infection Control and Medical Response National Emerging Infectious Diseases Laboratories

  1. Read More: Abstract

    The emergence of new infectious pathogens, particularly in resource limited settings, raises unique challenges such as bioethics of performing research during humanitarian crises, innovating in an environment where standard of care is shifting and providing access to care for the largest possible number of people. How can precision medicine aid in these challenges? Where does it fall short?


5:15 pm Conference Farewell: Networking Buffet