Agenda
September 22nd & 23rd
Cambridge, MA
All times listed below are in Eastern Daylight Time (EDT)
Complimentary breakfast with coffee bar.
Edritz Javelosa, PhD, CMT Research Foundation
Dr. Charlotte Sumner, a renowned expert in the field, will deliver the keynote talk highlighting the importance of collaboration in the development of new therapies in Charcot-Marie-Tooth (CMT).
Charlotte Sumner, MD, Johns Hopkins University
A conversation about the risk/benefit analysis of treatments, the symptoms that patients are most eager to resolve, pediatric considerations in CMT treatment, and exploring what meaningful treatments look like from the patients’ perspective.
Panelists: Sital Bhavsar, Eloise Schlafly, Javier Jover, Adrienne Weinstein, Michelle Moon, and Lily Sander
Sharon Hesterlee, PhD, EVP Chief Research Officer, Muscular Dystrophy Association will discuss the CMT drug pipeline and recent key advancements.
Sharon Hesterlee, PhD, Muscular Dystrophy Association
Examining the biggest challenges facing drug development for CMT.
Charles Abrams, MD, PhD, University of Illinois Chicago
A discussion of the common mechanisms of disease in CMT and insights into understanding and targeting these mechanisms in CMT research and therapy development.
Exploring the different models used in CMT research, and their strengths and limitations in advancing our understanding of the disease.
Animal Models: Rob Burgess, PhD, The Jackson Laboratory
The pros and cons of gene therapy in CMT, highlighting its potential for targeted treatment, disease modification, and long-term benefits, alongside challenges like delivery methods, immune responses, and the importance of personalized approaches based on specific genetic mutations.
Examining the progress and challenges in designing clinical trials and measuring efficacy.
Developed by Dr. Toby Ferguson’s team at Biogen, tofersen is a recently FDA-approved drug for ALS. Dr. Ferguson will provide a comprehensive overview of tofersen’s development, covering aspects such as pre-clinical work, animal studies, toxicology data, human experience, and clinical trials. The lessons learned from tofersen’s development provide insight into the development of treatments for CMT, showcasing shared connections and transferable insights between these neurodegenerative diseases.
Toby Ferguson, MD, PhD, Biogen
How academics can effectively prepare their asset(s) for licensing/acquisition, determine the right time to initiate the process, and deploy strategies to build a compelling business case for their project.
Panelists:
Charlotte Sumner, MD, Johns Hopkins University
Carol Greve- Philips, Biotech Independent Consultant
Seema Basu, PhD, Mass General Brigham
A look at biotech interest in CMT, the resources facilitating entry into the field, the challenges faced in raising funds for their companies, and the optimal timing for engaging larger companies or investors with their assets.
Panelists:
Steve O’Connor, PhD, Shift Pharmaceuticals
Thomas Neenan, PhD, Nanite
Meenu Chhabra Karson, Samsara Therapeutics
Douglas V Faller, MD, PhD, Oryzon Genomics
Arthur Suckow, PhD, Founder DTx Pharma, a Novartis company
Investors share insights on their interest in CMT, the risks and opportunities associated with the field, strategies to overcome those risks, and ways to stimulate increased investment and interest in CMT.
Panelists:
Megan Krench, PhD, Sanofi Ventures
Anela Vukoja, PhD, MSc, Apollo Health Ventures
Mark Veich, Deerfield Foundation
Complimentary breakfast with coffee bar.
In just five years The CMT Research Foundation has investigated scores of projects, funded 19, and completed initial projects with 6. Of those completed projects, 5 now have a clinical candidate. Here are some of the people who have been turning our mission from a theory into a highly successful reality.
Patrick Livney, CMT Research Foundation
Susan Ruediger, CMT Research Foundation
Arthur Suckow, PhD, DTx Pharma, a Novartis company
Peter deSilva, CMTRF Board Member
Brett McCray, MD, PhD, CMTRF Scientific Advisory Board
Bipasha Mukherjee-Clavin, MD, PhD, Johns Hopkins University
Jamal Hill, Paralympic Medalist
Rachel Salzman, DVM, Armatus Bio
Tara Haik CMT Research Foundation Ambassador
Paul August, CMTRF Scientific Advisory Board
Brian King, Violinist
Edritz Javelosa, PhD, CMT Research Foundation
A conversation with patients and caregivers about what CMT research advancements mean to them.
Panelists: Sital Bhavsar, Javier Jover, Adrienne Weinstein, Eloise Schlafly, Michelle Moon, Lily Sander
A patient/scientist covers crucial questions such as the reasons and considerations behind getting a genetic confirmation of your diagnosis, the process of obtaining genetic testing, the potential implications of receiving a genetic confirmation, and the financial issues, including who bears the cost of a genetic counselor.
Trisha Multhaupt–Buell, MS, Mass General Hospital
In-Person: Meet in private room for an opportunity to meet with an expert on your type of CMT.
Virtual: Discussion of CMT Types in one main virtual session.
Bruce Carter, PhD, Vanderbilt University
Type 2
Brett McCray, MD, PhD, University of Michigan
Type X
Κleopas A. Kleopa, MD, FAAN, FEAN, Cyprus Institute of Neurology and Genetics
Type 1B, Undiagnosed, and other types
Bipasha Mukherjee-Clavin, MD, PhD, Johns Hopkins University
Edritz Javelosa, CMT Research Foundation
A patient/scientist asks biotech executives about their projects, challenges, progress, and opportunities in advancing CMT therapies and how patients can participate in their work.
Sylvain Celanire, PhD, Augustine Therapeutics
Natalia Sacilotto, PhD, Oryzon Genomics
Meenu Chhabra Karson, Samsara Therapeutics
Christian Lorson, Shift Pharmaceuticals
Rachel Salzman, DVM, Armatus Bio
Why visit a CMT center? Learn what to expect during various diagnostic procedures and hear how patients can take proactive steps to participate and contribute to clinical trials for CMT research.
Κleopas A. Kleopa, MD, FAAN, FEAN, Cyprus Institute of Neurology and Genetics
A conversation led by a patient/scientist who will speak with a clinical trial expert on many of the questions patients want to ask about the clinical trial experience.
Riccardo Perfetti, MD, PhD, Applied Therapeutics
Members of the CMT community share the unique ways that they have gotten involved in becoming part of the solution for CMT.
Panelists: Lily Sander, Miron Hall, Anushka Noori, Shelley Reid, and The Stahl Family