Agenda
September 26-28, 2024
Cambridge, MA
All times listed below are in Eastern Daylight Time (EDT)
Kathrin Meyer, PhD, Assistant Professor, Physical Medicine and Rehabilitation, University of Missouri School of Medicine and Chief Scientific Officer, Alcyone Therapeutics
Charles Abrams MD, PhD, Professor, Department of Neurology and Rehabilitation, University of Illinois, Chicago
Development of a Novel Neuromuscular Organoid Model for Disease Modeling
Rob Prior, PhD, Postdoctoral Researcher, University of Bonn, Germany
Small Molecule Modulators of a PRAF2 Complex Regulate PMP22 Proteostasis with Potential Therapeutic Benefit in CMT-1A
Tim Piser, PhD, Head of R&D, Samsara Therapeutics
A Mouse Model for ITPR3 Related CMT1 and Evaluation of ASO-induced Exon Skipping
Adriana Rebelo, PhD, Scientist, University of Miami, Miller School of Medicine
Novel Charcot-Marie-Tooth Type 2E (CMT2E) mouse models of Neurofilament Light (Nefl) E396K: Phenotypic Validation and Therapeutic Analysis
Dennis Perez-Lopez, PhD Candidate, University of Missouri
Application of Wearable Sensors to Assess Disease Severity in Adults with Charcot-Marie-Tooth Disease
Kayla Cornett, PhD, Postdoctoral Researcher, University of Sydney and Sydney Children’s Hospital Network
Complimentary Breakfast and Coffee Bar.
Carsten Bönnemann, MD, Senior Investigator and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section in the Neurogenetics Branch of the National Institute of Neurological Disorders and Stroke, National Institue of Health
Moderator: Bruce D. Carter, PhD, Professor of Biochemistry, Vanderbilt University
Speakers:
You Can’t Fix What you Don’t Understand: Understanding Basic Biological Mechanisms to Identify Therapeutic Targets in CMT
Rob Burgess, PhD, Professor, Rare Disease Translational Center, The Jackson Laboratory
Discovery of Small Molecules that Alter the Trafficking of PMP22 as a Potential Route to CMT1A, CMT1E AND HNPP Therapies
Charles Sanders, PhD, Professor of Biochemistry & Medicine, Vice Dean of Basic Sciences and Associate Dean for Research, Vanderbilt University
Myotubularin-Related (MTMR) Phosphoinositide Phosphatases and Inherited Neuropathies
Alessandra Bolino, PhD, Group Leader, Human Inherited Neuropathies, Ospedale San Raffaele
Moderator: Bruce D. Carter, PhD, Professor of Biochemistry, Vanderbilt University
Speakers:
TRPV4 Inhibition in the Treatment of CMT2C
David Goldstein, PhD, Co-Founder and CEO, Actio Biosciences
NMD670, a Chloride Channel 1 (ClC-1) Inhibitor, a Novel Potential Therapeutic Alternative for Charcot-Marie-Tooth Disease
Jorge A. Quiroz, MD, MBA, Executive Vice President, Chief Medical Officer, NMD Pharma
Developing Best-in-Class HDAC6 Inhibitors for the Treatment of CMT1A
Frederik Rombouts, PhD, Vice President, Drug Discovery, Augustine Therapeutics
Activation of the IRE1/Xbp1 Pathway of the UPR Ameliorates Disease Parameters in Models of Proteotoxic CMT1B
Maurizio D’Antonio, PhD, Group Leader Biology of Myelin Unit, Ospedale San Raffaele
Moderator: Kathrin Meyer, PhD, Assistant Professor, Physical Medicine and Rehabilitation, University of Missouri School of Medicine and Chief Scientific Officer, Alcyone Therapeutics
Speakers:
Current Opportunities and Challenges for CMT Gene Therapies
Kleopas Kleopa, MD, PhD, FAAN, FEAN, Professor and Senior Neurologist, The Cyprus Institute of Neurology and Genetics
Supporting Translation of the CMT1A Gene Therapy Through AAV-Mediated Silencing of PMP22 in Mice and Non-Human Primates
Scott Harper, PhD, Principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute, Nationwide Children’s Hospital
Development of a Knockdown and Replace Gene Therapy for CMT1B
Afrooz Rashnonejad, MSc, PhD, Principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute, Nationwide Children’s Hospital
Development of Gene Replacement Therapy for CMT4A Demyelinating Neuropathy
Xin Chen, PhD, Assistant Professor, UT Southwestern Medical Center
The Development of Tissue Tropic AAVs – Considerations Inside Out
Guangping Gao, Principal Investigator, UMass Chan Medical School and Co-founder, Voyager Therapeutics
Developing Lipid Nanoparticles for non-Liver RNA Delivery
Ryan Zenhausern, PhD Candidate, Georgia Tech and Emory University
Moderator: Charles Abrams MD, PhD, Professor, Department of Neurology and Rehabilitation, University of Illinois, Chicago
Speakers:
Lessons Learned From ALS Clinical Development: Opportunities for CMT Drug Development
Toby Ferguson, MD, PhD, Chief Medical Officer, Voyager Therapeutics
Current Status of Clinical Outcome Assessments and Biomarkers in CMT
Brett McCray, MD, PhD, Assistant Professor in Neurology, University of Michigan
Govorestat for CMT-SORD: Designing a Clinical Trial for a Slow Progressing CMT
Riccardo Perfetti, MD, PhD, Chief Medical Officer, Applied Therapeutics
FDA Efforts to Advance Medical Product Development for Rare Diseases
Celia Witten, MD, PhD, Deputy Director, Center for Biologics Evaluation and Research, Food and Drug Administration
Moderator: Arthur Suckow, PhD, Founder of DTx Pharma
Panelists:
Gerhard Koenig, PhD, Executive Chairman, Augustine Therapeutics
Ilan Ganot, MBA, CEO, Alesta Therapeutics
David Goldstein, PhD, Co-Founder and CEO, Actio Biosciences
Tim Piser, PhD, Head of R&D, Samsara Therapeutics
Rachel Salzman, DVM, CEO, Armatus Bio
Adrienne Weinstein, Endgame Campaign Committee Member, CMTRF
Complimentary Food and Beverages
Patient Community Day
Saturday, September 28th
Peter deSilva, Board Chair, CMT Research Foundation
Jamal Hill, Paralympic Medalist and CMT Patient