We are thrilled to announce our collaboration and sponsored research with The Meisler Lab at the University of Michigan!
The Meisler Lab has long been known for its work and interest in rare diseases. Dr. Miriam Meisler and Dr. Guy Lenk have been studying the FIG4 gene involved in CMT4J for over a decade. Their work continues to provide the foundation of scientific knowledge for a gene therapy clinical trial and other treatments for CMT4J.
Because of your support, we are able to fund the University of Michigan a $125,000 research grant focusing on fast-tracked development of meaningful therapeutics for people with CMT4J.
THREE SEPARATE RESEARCH TRACKS INCLUDE:
- ASO Pathways for FIG4: ASO is short for antisense oligonucleotide—say that three times fast! In a nutshell, ASO’s are a sort of synthetic band-aid that helps to fix the mixed-up genetic code caused by a mutation. Sometimes, by skipping over the damaged part of a gene, or making another part work better, the missing protein can be made to work again. Recent, successful examples of ASO’s include a drug called Spinraza that has brought lifesaving, muscle-saving benefits to people with SMA (spinal muscular atrophy), a neuromuscular disease quite similar to CMT4J. Another example is Exondys, a drug made for people with a certain type of Muscular Dystrophy. Exondys has been shown to help stave off respiratory decline in this patient community and is in on-going studies to show improvement in mobility and muscle strength. Our goal is to develop a novel ASO for the FIG4 gene and bring it forward as a treatment in less than a year!
- Drug-Repurposing: CureCMT4J and the University of Michigan already work together to identify drugs that might be used for similar diseases that might also have an impact on CMT4J. Our grant to UMich will enable us to explore more targets, conduct research and test some of these treatments.
- Novel, alternative lysosomal protein identification and testing for FIG4 rescue research approaches: As a forward-looking approach, we aim to identify more efficient pathways for future CMT4J therapeutics. These will help to bring down both the time and cost of manufacturing gene therapies and other treatments.
Lastly, CureCMT4J’s grant to the University of Michigan enabled the recruitment of another post-doc researcher to help fast-track our research quickly.
“It is very meaningful to us that our many years of research on FIG4 and CMT4J have reached a point at which medical applications are on the near horizon.”–Dr. Miriam Meisler, Director of the Meisler Lab, University of Michigan
CMT4J AAV9 GENE THERAPY TRIAL
While the loss of loved ones and lives throughout the world is the over-arching stress and sadness during a global pandemic, another heartbreaking story has been the halt of all non-covid related research.
The rare disease community has been hit particularly hard by this, with clinical trials and preclinical studies abandoned without a moment’s notice. While the pandemic has certainly affected our overall timelines, we feel fortunate to share that our research, deemed critical, has continued to move forward. We are so grateful for our dedicated scientists.
We will keep you informed with updates regarding a date for a clinical trial and the resumption of our CMT4J Natural History Study.
Thank you for your continued support! we can’t do any of this without you!