Full Program
RE(ACT) CONGRESS AMERICA 2019:
WEDNESDAY, May 8th
Special morning sessions 9 to 12 open to all:
Parallel session 1: Multi-national clinical trials/registries – Chairs: 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 A 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”
- Kathleen Reape, USA “Clinical and Regulatory Considerations and the U.S. Approval of LUXTURNA™ (voretigene neparvovec-rzyl)”
Lunch 12 to 13
POSTER SESSION D & E 13 to 14
E. Collaborative Session (CORD, RDI, CIHR, CADTH, CHEO, NCATS, FDA, EMA, UDNI, IRDiRC) Afternoon 14 to 17 “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
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 11, 2019
