The Power of Partnership in CMT Gene Therapy Research

Video

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the origins of the CMTRF.

“When my partner and I started the research foundation in 2018, we knew that there was opportunity for us to bring more money, partners, investigators, efforts, and more biotechs and pharma into the CMT space. We knew that if we organized our effort, that there would be an opportunity for us to do that.”

The CMT Research Foundation (CMTRF)’s mission is to help researchers find treatments for Charcot-Marie-Tooth disease (CMT). The foundation funds promising projects, including cell and gene therapy approaches, to address unmet needs in CMT.

In line with their goal, the CMTRF recently signed an agreement with Nationwide Children’s Hospital and awarded a $500,000 grant to Afrooz Rashnonejad, PhD, principal investigator, Nationwide and Assistant Professor, Department of Pediatrics, Ohio State University. Rashnonejad’s research is taking a knock down and replace approach to silencing the mutated MPZ gene and replacing it with a functioning gene.

CGTLive spoke with Susan Ruediger, founder and chief mission officer, CMT Research Foundation, to learn more about the research foundation and its origins. She discussed the importance of partnering between organizations, academia, and industry as well as an early partnership with DTx Pharma that demonstrated the power of partnering.

REFERENCE
CMT Research Foundation Awards Grant to Nationwide Children’s Hospital’s Dr. Afrooz Rashnonejad to Develop Gene Therapy to Cure or Alleviate CMT1B. News release. CMTRF. July 18, 2022. https://cmtrf.org/nationwide-childrens-hospital-cmt1b-press-release/
Recent Videos
Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
Daniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization
Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Barry J. Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Related Content
© 2024 MJH Life Sciences

All rights reserved.