Agenda

September 22nd & 23rd Cambridge, MA
All times listed below are in Eastern Daylight Time (EDT)

Scientific Day

Friday, September 22nd

7:00 AM Breakfast & Networking

Complimentary breakfast with coffee bar.

8:00 AM Welcome
Cleary Simpson, CMT Research Foundation
Edritz Javelosa, PhD, CMT Research Foundation
Charles Abrams, Scientific Programming Committee Chair
8:10 AM Keynote Address

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

8:40 AM Patient Fireside Chat- Life with CMT

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.

Moderator: Susan Ruediger, CMT Research Foundation
Panelists: Sital Bhavsar, Eloise Schlafly, Javier Jover, Adrienne Weinstein, Michelle Moon, and Lily Sander
9:20 AM Overview of the CMT Drug Development Pipeline

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

9:40 AM Challenges in Drug Development

Examining the biggest challenges facing drug development for CMT.

Charles Abrams, MD, PhD, University of Illinois Chicago

9:50 AM Common Mechanisms of Disease

A discussion of the common mechanisms of disease in CMT and insights into understanding and targeting these mechanisms in CMT research and therapy development.

Axonal Degeneration: Jeff Milbrandt, MD, PhD, Washington University St. Louis 
Stress Response: Rick Dobrowsky, PhD, University of Kansas
Mitochondrial Dysfunction: Albert Misko, MD, PhD, Novartis
Inflammation: Dennis Klein, PhD, University Hospital Wuerzburg
 
11:10 AM Models of CMT

Exploring the different models used in CMT research, and their strengths and limitations in advancing our understanding of the disease.

iPSc Models: Gabsang Lee, PhD, DVM, Johns Hopkins University
Animal Models: Rob Burgess, PhD, The Jackson Laboratory
Human on a Chip Models: James Hickman, PhD, University of Central Florida
 
12:10 PM The State of Gene Therapy in CMT

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.

Afrooz Rashnonejad, MSc, PhD, Nationwide Children’s Hospital
 
12:30 PM Lunch (Sponsored by Armatus Bio) & Poster Viewing Session
1:20 PM Clinical Trial Readiness

Examining the progress and challenges in designing clinical trials and measuring efficacy. 

Trial Design Best Practices: Kevin Flanigan, MD, Nationwide Children’s Hospital
Clinical Trial Readiness: Katy Eichinger, PT, PhD, NCS, University of Rochester
Biomarkers: Mario Saporta, MD, University of Miami
2:30 PM Break
3:00 PM Translating Academic Science to Business: So you think you have a promising asset- how to build the bridge from bench to market?

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.  

Moderator: Margaret Read, PhD, Vanderbilt University
Panelists:
Charlotte Sumner, MD, Johns Hopkins University
Carol Greve- Philips, Biotech Independent Consultant
Seema Basu, PhD, Mass General Brigham
H.C. Huang, PhD, Deerfield Discovery and Development (3DC)
3:50 PM Pushing Boundaries in CMT Research: Early-Stage Biotech Companies' Evolution and Insights on Therapy Development

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.  

Moderator: William Motley, MD, PhD Rapport Therapeutics
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
Sylvain Celanire, PhD, Augustine Therapeutics

 

4:50 PM Rare Disease Drug Development: Perspectives from Investors on Opportunities and Challenges

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.  

Moderator: Matt Hammond, PhD, MBA, RA Capital Management
Panelists:
Megan Krench, PhD, Sanofi Ventures
Anela Vukoja, PhD, MSc, Apollo Health Ventures
Mark Veich, Deerfield Foundation
 
5:35 PM Poster Award
Charles Abrams, MD, PhD and the Scientific Programming Committee
5:45 PM Scientific One on One Sessions
Invite only
7:00 PM Cocktail Reception

Patient Community Day

Saturday, September 23rd

8:00 AM Breakfast
Complimentary breakfast with coffee bar.
9:00 AM Kick-Off Session

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.

Cleary Simpson, CMT Research Foundation
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
 
10:00 AM Patient Voices

A conversation with patients and caregivers about what CMT research advancements mean to them.

Moderator: Susan Ruediger, CMT Research Foundation
Panelists: Sital Bhavsar, Javier Jover, Adrienne Weinstein, Eloise Schlafly, Michelle Moon, Lily Sander 
 
10:45 AM Understanding the Importance of Genetic Testing

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.

Moderator: Wolfgang Pernice, PhD, Columbia University
Trisha MulthauptBuell, MS, Mass General Hospital
Nicole Faulkner, PhD, FACMG, Invitae 
 
11:45 AM Lunch session on CMT Types (Sponsored by Samsara Therapeutics)

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.

Type 1A, 1E and HNPP (Disease of PMP22) 

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

All types: Virtual 
Mario Saporta, MD, PhD, MBA, FAAN, University of Miami Miller School of Medicine
Rob Prior, PhD, University of Bonn
 
 
1:15 PM The Impact of the CMT Research Foundation

Edritz Javelosa, CMT Research Foundation

 

1:30 PM Patient-Powered Perspectives: Biotech Insights on CMT and Collaborating with Patients

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.

Moderators: Allison Taylor, Johns Hopkins University
Sylvain Celanire, PhD, Augustine Therapeutics
Natalia Sacilotto, PhD, Oryzon Genomics

Meenu Chhabra Karson, Samsara Therapeutics
Christian Lorson, Shift Pharmaceuticals
Rachel Salzman, DVM, Armatus Bio

 

2:45 PM Taking you inside the Clinical Trial Process, Part I: The Scientific Perspective

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  

3:15 PM Taking you Inside the Clinical Trial Process, Part II: The Patient Perspective

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.

Moderator: Kathryn Moss, PhD, Johns Hopkins University 

Riccardo Perfetti, MD, PhD, Applied Therapeutics 

3:45 PM Break
4:00 PM Becoming Part of the Solution

Members of the CMT community share the unique ways that they have gotten involved in becoming part of the solution for CMT.

Moderators: Anna Combes, CMT Research Foundation and Chelsea Layton, CMT Research Foundation

Panelists: Lily Sander, Miron Hall, Anushka Noori, Shelley Reid, and The Stahl Family 

4:30 PM Catalyst Award Announcement
4:40 PM Networking
5:30 PM Sunset Riverboat Cruise on the Charles River