The Power of Partnership in CMT Gene Therapy Research

“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/