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RE(ACT) CONGRESS AMERICA 2019:

Download here the Congress Abstract Book

WEDNESDAY, May 8th

Special morning sessions 9 to 12 open to all:

Parallel session 1: Multi-national clinical trials/registries – Chair: Dr Anne Junker (Maternal Infant Child and Youth Research Network)

Panellists:

  • Brendan Lee, USA “Brittle Bone Disorders Consortium: The skeletal system as a target and model for rare disease research”
  • Sabine Kläger, FR “Challenges, considerations and support for investigator initiated, multi-national trials across Europe – ECRIN”
  • Beth Potter, CAN“Generating evidence to improve care and outcomes for pediatric inherited metabolic disease: the Canadian Inherited Metabolic Diseases Research Network”

Parallel session 2: Generalizable therapeutic approaches for RD – Chair Pr. Chris McMaster (Dalhousie University)

Panellist: 

  • James Dowling, CAN “Developing therapies for congenital myopathies:  unmet needs and potential successes”
  • Hanns Lochmuller, DE “Targeted therapies for rare neuromuscular disorders – first steps towards a treatabolome”
  • Anne E. Carpenter, USA “Targeting Disease via Cell Painting and Image-Based Profiling”

A. Session, Afternoon 14 to 17 “Gene Editing”

  • Special talk: Aled Edwards, CAN “Open science drug discovery – the scientific case and an operating business model”
  • Dwi Kemaladewi, CAN “Interventional genomics in congenital muscular dystrophy”
  • Jacques P. Tremblay, CAN “Development of therapies for hereditary diseases by modifying genes with the CRISPR/Cas9 technology”
  • Lauryl Nutter, CAN “Genome editing to produce precision models for precision medicine”

POSTER SESSION 17 to 18

Public Opening Ceremony 18 to 20 

THURSDAY, May 9th

B. Session, Morning 9 to 12
 “Cell & Gene Therapy“

  • Alberto Auricchio, IT “AAV gene therapy from bench to bedside”
  • William Stanford, CAN “Preclinical stem cell models of tuberous sclerosis complex and its related lung neoplasm lymphangioleiomyomatosis for therapeutic development”
  • Stephanie Cherqui, USA “Hematopoietic Stem Cell Gene Therapy for Cystinosis: Clinical Translation and Mechanism of Action”
  • Marina Cavazzana, FR “Gene therapy for inherited diseases of the hematopoietic system”

Lunch 12 to 13

POSTER SESSION B & C 13 to 14

C. Session, Afternoon, 14 to 17 “Indigenous populations”

  • Laura Arbour, CAN “Rare diseases in Indigenous populations: Where do we start?”
  • Gareth Baynam, AUS “Life Languages”
  • Victoria Siu, CAN “Genetic Disorders in the Amish and Mennonite communities: Harnessing the results of research to benefit child health”
  • Maui  Hudson, NZ “The Responsibility of Researching Rare Disorders & Rare Populations”
  • Cheryl Rockman-Greenberg, CAN “From gene discovery to newborn screening in Indigenous populations”

FRIDAY, May 10th

D. Session, Morning 9 to 12
 “Stem Cells/Regenerative Medicine/Genetically Modified Cell Therapies/Clinical Applications of Gene Therapies”

  • Tony Rupar, CAN “Gene therapy in Lysosomal storage diseases – Fabry disease and Metachromatic Leukodystrophy”
  • Ian  MacDonald, CAN “Ocular gene therapy: what are we learning from clinical trials”
  • Monica Justice, CAN “Therapeutic insights for Rett syndrome from the study of MECP2 modifiers in mice”
  • James J Hickman, USA “Building human-on-a-chip phenotypic models to predict in vivo outcomes
    for efficacy and toxicity for rare diseases”
  • Tejashri Purohit-Sheth, USA ” FDA Expedited Pathways and Regenerative Advanced Therapy Designation”

Lunch 12 to 13

POSTER SESSION D & E 13 to 13:30

E. Collaborative Session (CORD, RDI, CIHR, CADTH, CHEO, NCATS, FDA, EMA, UDNI, IRDiRC) Afternoon 13:30 to 1:307 “Patient focused drug development“

  • Marlene Haffner, USA “I am a Patient. Please Listen to ME”
  • Nick Sireau, UK “Developing a treatment for the ultra rare Black Bone Disease”
  • Larissa Lapteva, USA “Patient-Focused Development of Medical Products: Regulatory Perspective”
  • Calvin N. Ho, USA “How Advocates Can Bring Patient Experience Data to Regulators and Industry”
  • Soo-Kyung Lee, USA “What Does the FOX Say?  FOXG1 Orchestrates Cortico-Cortical Connections”
  • Keith Massey, CAN “Riboflavin Transporter Deficiency: Clinical Presentation, Genetics and Treatment “

SATURDAY and SUNDAY, May 11th &12th
 (Not included in the registration, you need to register here)

Rare Disease International (RDI) & Canadian Organization for Rare Disorders (CORD): “A Rare International Dialogue” (draft program) – A total of 20% discount will be offer if you register to both meetings.

Program updated April 23, 2019

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  • About
  • Attendees
    • Venue
    • Toronto
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    • Full Program
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