Our CMT Research Approach & Priorities
The mission of the CMT Research Foundation is to deliver treatments and cures for CMT during our lifetime. The CMT Research Foundation pursues this goal by identifying and bridging the gaps in biomedical research, with a focus on areas that are most likely to result in clinical treatments.
We are most focused on translational research, but also fund discovery phase research with a clear link to speeding progress toward treatments and cures.
We invest in the most promising Charcot-Marie-Tooth research that will best achieve treatments and a cure, and create strategic alliances within the academic, biotech, pharmaceutical and regulatory communities to accelerate progress.
We fund projects with the highest likelihood of leading to clinical trials and approved treatments in the near term. All investments are gated, meaning projects need to achieve milestones defined for each phase of work before receiving funding for the next.
Research proposals are carefully vetted by the CMTRF’s Scientific Advisory Board. The Board evaluates each proposal based on the excellence of the investigator, the work proposal, as well as the project’s ability to be translated into treatments for people with CMT. Because we feel a sense of urgency to deliver important scientific breakthroughs, proposals are quickly, but comprehensively, vetted.
We forge partnerships that include a revenue share program, which will in turn be reinvested into translational research for treatments and cures for all types of CMT. Every contract includes a return on the Research Foundation’s initial investment which is then reinvested in new projects.
Research Priorities
Advancing Gene Therapy
- Control the expression of genetic causes of CMT through gene editing, silencing, epigenetics, splice modification, and other approaches, including both gene therapy and small molecule approaches.
- Understand and control modifier genes that impact disease severity and/or progression in CMT.
Promoting Nerve Health
- Understand and pharmacologically control cellular and molecular pathways that affect axon health, including survival, regeneration, and target connectivity.
- Understand and pharmacologically control cellular and molecular pathways that affect peripheral myelination, including Schwann cell survival and function.
Overcoming The Delivery Challange
- Understand and manipulate the peripheral nerve barriers
- Develop novel delivery mechanisms.
- Refine existing delivery mechanisms.
De-Risking Clinical Trials (Secondary Priority)
- Increase the rigor of CMT research with an eye toward enhancing its drug development value.
- Expand data and sample sharing in CMT research.
- Define the health economics of CMT, including global incidence/prevalence, economic burden and effect of therapeutics.
- Develop existing or novel outcome measures and biomarkers that reliably measure and predict disease progression in order to support and accelerate clinical trials.
Research We Fund
Transparency is important to us. We provide detailed project descriptions about the CMT disease and treatment research we support.
Clinical Trials
Use our clinical trial finder to find out if you qualify to participate in currently available CMT therapy trials.
Apply for Funding
CMTRF only funds projects that will lead toward approved drugs for CMT.
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