Agenda
September 16 & 17th
Cambridge, MA
All times listed below are in Eastern Daylight Time (EDT)
7:00-8:00 am Breakfast
- Complementary breakfast with coffee bar
OPENING SESSIONS
8:00-8:15 am Welcome Session
- Cleary Simpson, CMT Research Foundation
- Keith Fargo, PhD, CMT Research Foundation
- Marina Kennerson, PhD, ANZAC Research Institute ad Sydney Medical School
8:15-8:45 am Keynote Address
- Chris Austin, MD, Flagship Pioneering and Vesalius Therapeutics
8:45-9:15 am Recent Development in CMT Research
- Dr. Vera Fridman, MD, University of Colorado
9:15-9:30 am Break
THEME SESSIONS
9:30-11:30am Metabolism and Axon Degeneration
Moderator: Edritz Javelosa, PhD, Muscular Dystrophy Association
- Metabolic coupling between Schwann cells and axons- Elisabetta Babetto, PhD, University at Buffalo
- SORD deficiency- Andrea Cortese, MD, PhD, University College London Queen Square Institute of Neurology
- Mitochondrial dysfunction and axon transport- Alexander Van Der Bliek, PhD, UCLA School of Medicine
- SARM1 in CMT1A- Kathyrn R. Moss, PhD, Johns Hopkins University
11:30-12:30 pm Lunch Buffet
12:30-2:00 pm Genetic Medicine
Moderator: Alessandra Bolino, PhD, San Raffaele Scientific Institute
- Gene replacements for GAN and CMT4J – Rachel Bailey, PhD, University of Texas Southwestern Medical Center
- PMO ASOs for CMT1A – Christian Lorson, PhD, University of Missouri and Shift Pharmaceuticals
- SMARD1/CMT2S Gene Therapy: Benchside to Bedside- Shibi Likhite, PhD, Nationwide Children’s Hospital
2:00-2:30 pm Break
2:30-5:00 pm Small Molecule Approaches
Moderator: Paul August, PhD, ReviR Therapeutics
- Inflammation in Type 1 CMT – Rudolf Martini, PhD, University of Würzburg
- Modulating the unfolded protein response – Maurizio D’Antonio, PhD, Ospedale San Raffaele
- Mitofusin activators for CMT2A – Gerald Dorn, MD, Washington University in St. Louis and Mitochondria in Motion
- PIKfyve inhibition for CMT4B1 – Alessandra Bolino, PhD, San Raffaele Scientific Institute
- Targeting autophagy for CMT1A – John Blackwood, PhD, Samsara Therapeutics
5:00-5:30 pm Break
5:30-6:30 Biomarkers for CMT
Moderator: Tiina Urv, PhD, NIH National Center for Advancing Translational Sciences
- Muscle fat fraction – Jasper Morrow, MBChB, PhD, University College London Queen Square Institute of Neurology
- An international collaboration to discover CMT biomarkers (NCAM1 and GDF15)- Matt Jennings, PhD, Columbia University
6:30-7:00 Closing Session
8:00-9:00 am Breakfast
9:00-9:20 am Kick-Off Session
- Cleary Simpson, CMT Research Foundation
- Pat Livney/Susan Ruediger, CMT Research Foundation
- Keith Fargo, PhD, CMT Research Foundation
9:20-10:35 am Unlocking the Genetic Secrets of CMT
Moderators: Marina Kennerson, PhD, ANZAC Research Institute and University of Sydney; and Wolfgang Pernice, PhD, Columbia University
- Jim Lupski, MD, PhD, Baylor College of Medicine
- Vincent Timmerman, PhD, University of Antwerp
- Stephan Zuchner, MD, PhD, University of Miami Miller School of Medicine
- Andrea Cortese, MD, PhD, University College London Queen Square Institute of Neurology
- Anthony Antonellis, PhD, University of Michigan
10:35-10:45 am Break
CLINICAL TRIALS
10:45-11:15 am Understanding Clinical Trials
- Melissa DuPont, Sanofi
11:15-11:45 am Clinical Trial Readiness: What are Natural History Studies?
- David Herrmann, MBBCh, University of Rochester
LUNCH AND BREAKOUT SESSSIONS
11:45-12:00 pm Break
- Grab lunch before heading into breakout sessions
12:00-12:45 pm Breakouts Round 1
Small group discussions for each topic, led by key clinicians in CMT.
- Types 1A and 1B – Kathryn R. Moss, PhD, Johns Hopkins University; and David Herrmann, MBBCh, University of Rochester
- Type 2A – Mario Saporta, MD, PhD, MBA, University of Miami Miller School of Medicine
- Type X – In Person: Charles Abrams, MD, PhD, University of Illinois at Chicago (UIC); Virtual: Wolfgang Pernice, PhD, Columbia University; and Robert Prior, PhD, University of Bonn
- Other Types and ‘I Don’t Know My Type’ – Reza Seyedsadjadi, MD, Massachusetts General Hospital and Harvard Medical School
12:45-12:55 pm Break
- Switch to Round 2 Breakout Sessions
12:55-1:40 pm Breakouts Round 2
- Genetic Testing – Marina Kennerson, PhD, ANZAC Research Institute and University of Sydney; and Wolfgang Pernice, PhD, Columbia University
- How Can YOU Make an Impact on CMT – Chelsea Layton, CMT Research Foundation
- Bracing and Orthotics: What You Need to Know – Ken Cornell, CO, Cornell Orthotics & Prosthetics
- Pediatrics: Care and Research – Leslie Hotchkiss Hayes, MD, Boston Children’s Hospital and Harvard Medical School
1:40-1:50 pm Break
- Switch back to main conference room
AFTERNOON SESSIONS
1:50-2:30 pm CMT Clinical Trials: What's New and What's Next
- Dr. Mario Saporta, MD, PhD, MBA, University of Miami Miller School of Medicine
2:30-3:45 pm Biopharma Activity in CMT
Moderator: Paul August, PhD, ReviR Therapeutics
- Arthur Suckow, PhD, DTx Pharma
- Scott Q. Harper, PhD, Armatus Bio
- Thomas Pedersen, PhD, NMD Pharma
- Sylvain Celanire, PhD, Augustine Therapeutics
- Christian Lorson, PhD, Shift Pharma
3:45-4:30 pm Closing Session
4:30-5:30 pm Networking With the Speakers
5:30-7:00 pm Sunset Riverboat Cruise on the Charles River
Explore the beautiful Charles River with the team at the CMT Research Foundation and the speakers from the weekend. This is a great opportunity to get to know everyone working in CMT personally.
Seating is limited – please reach out to [email protected] to request your seat(s)!