Sep 18, 2018 - Introduction - James Foster, Charles River. 9:00 AM - 9:30 AM. 2018 Award Winner - David Hysong, Shepherd Therapeutics. 9:30 AM - 10:15 ...
LESSONS LEARNED FROM RARE DISEASE AND PERSONALIZED MEDICINE 2ND ANNUAL CHARLES RIVER WORLD CONGRESS
CAMBRIDGE, MA SEPTEMBER 17-18, 2018
Monday, September 17, 2018 8:30 AM - 9:00 AM
Introduction - James Foster, Charles River 9:00 AM - 9:30 AM
2018 Award Winner - David Hysong, Shepherd Therapeutics 9:30 AM - 10:15 AM
Keynote - The Cystic Fibrosis Foundation’s Venture Philanthropy Model, Preston Campbell 10:15 AM - 10:45 AM
Orphan to IND, The Jain Foundation’s Efforts Toward Making a Rare Disease Attractive to Drug Companies Doug Albrecht, Jain Foundation 10:45 AM - 11:00 AM - Morning Break - Day 1 11:00 AM - 11:30 AM
Beyond Clinical Trials: Reshaping Drug Development with Expanded Access and Real-World Data Martin Naley, MyTomorrows 11:30 AM - 12:00 PM
Panel Discussion - Surfacing the Best Model for Drug Discovery, Moderated by Antti Nurmi, Charles River 12:00 PM - 1:00 PM - Networking Lunch - Day 1 1:00 PM - 1:30 PM
Is Open Science a Viable Option for Discovering, Developing, and Commercializing Drugs for Orphan Diseases? Owen Roberts, Meds4Kids Pharma 1:30 PM - 2:00 PM
The Development of Substrate Reduction Drugs for the Treatment of Glycosphingolipidoses James Shayman, University of Michigan 2:00 PM - 2:15 PM - Afternoon Break - Day 1 2:15 PM - 2:45 PM
Panel Discussion - You Have Your Drug Target, Now What? Moderated by Martin O’Rourke, Charles River 2:45 PM - 3:15 PM
Technobites - Technologies to Facilitate Drug Development Dynamic Cell Signaling Monitoring John Groten, Pamgene & Functional Ultrasound, Ludovic Lecointre, Iconeus 3:15 PM - 4:00 PM
Mighty Voices: Harnessing the Power of Patients and Caregivers, Mike Porath, TheMighty.com 4:00 PM - 4:15 PM - Closing Remarks - Day 1 4:30 PM - 6:00 PM
Networking Reception - My Reason Why
LESSONS LEARNED FROM RARE DISEASE AND PERSONALIZED MEDICINE 2ND ANNUAL CHARLES RIVER WORLD CONGRESS
CAMBRIDGE, MA SEPTEMBER 17-18, 2018
Tuesday, September 18, 2018 8:45 AM - 9:00 AM
Introduction - James Foster 9:00 AM - 9:30 AM
Update from 2017 Award Winners - The Path to Prevention of Prion Disease Sonia Vallabh & Eric Minikel, Prion Alliance/Broad Institute of MIT and Harvard 9:30 AM - 10:15 AM
Keynote - AFM-Téléthon: A Game Changer Patient Organization in the World of Medical Research Serge Braun, PharmD, PhD, AFM-Téléthon 10:15 AM - 10:45 AM
Using a Collaborative Team Approach to Accelerate Towards Effective Treatments for Rare Diseases Jill Weimer & Antti Nurmi, Sanford Health & Charles River 10:45 AM - 11:00 AM - Morning Break - Day 2 11:00 AM - 11:30 AM
Phenotypic Screening as a Source of Novel Molecules, Targets and Mechanisms in Neurodegenerative Disease Ken Rhodes, Yumanity 11:30 AM - 12:00 PM
Panel Discussion - The Ecosystem of Foundations, Academics, Industry, and CRO Moderated by Anjli Venkateswaran, Charles River 12:00 PM - 1:00 PM - Networking Lunch - Day 2 1:00 PM - 1:30 PM
Human Stem Cell-Derived Adeno-Associated Viruses Are Highly Efficient Vectors for Both Gene Transfer and Nuclease-Free Gene Editing Jeff Ellsworth, Homology Medicine 1:30 PM - 2:00 PM
Identification of a Novel Kinase Target for Myotonic Dystrophy Therapy J. David Brook, University of Nottingham 2:00 PM - 2:30 PM
A Non-Profit Mechanism for Moving Proof-of-Concept Studies to Clinical Therapies for Rare Retinal Disorders Scott Dorfman, Odylia Therapeutics 2:30 PM - 2:45 PM - Afternoon Break - Day 2 2:45 PM - 3:15 PM
Panel Discussion - Overcoming Technical Challenges to Drug Discovery, Moderated panel discussion 3:15 PM - 4:00 PM
Finding Tomorrow - One Family’s Rare Disease Journey from a Bake Sale to an Approved Therapy Mark Dant, EveryLife Foundation 4:00 PM - 4:15 PM - Closing Remarks - Day 2
