CMTA is funding researchers at La Jolla Labs, working with John Svaren, PhD, to develop an ASO treatment that increases MFN1 expression as a potential way to compensate for the damaging effects of MFN2 mutations that cause CMT2A.
CMTA Invests $110,500 in Novel ASO Medicine for CMT2A
The Charcot-Marie-Tooth Association announced a $110,500 investment partnering with La Jolla Labs to develop an antisense oligonucleotide medicine targeting CMT2A, the most common axonal form of CMT, using a novel approach to increase MFN1 expression.
A Backup Gene Offers a New Path to CMT2A Treatment
What if the answer to CMT2A is already in your cells? A new CMTA partnership is testing whether switching on a backup gene could protect nerves when MFN2 fails.
Is Spinal Fluid the Way to Gene Therapy in CMT?
Most kinds of CMT come from a problem in a single gene. Repairing that faulty...
CMTA Leading the Way in CMT1A
CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments.
CMTA-STAR Delivery Science: Four Approaches for CMT
CMTA is funding four research projects targeting a fundamental barrier to CMT treatment: getting therapies past the biological defenses protecting peripheral nerves. The $523,000 CMTA-STAR investment supports complementary delivery approaches, including gene editing, RNA medicine, and nanoparticles, across multiple CMT subtypes.
Peptide-LNPs Deliver siRNA Against PMP22 in CMT1A
With combined support totaling $102,968 from CMTA, Shark Tooth Bio, and the National Research Council...
Blood–Brain Barrier-Crossing Conjugates for Treating CMT
CMTA-funded research at Mount Sinai is studying how RNA medicines can be delivered into nerve cells in CMT, overcoming biological barriers that block treatment.
STEP Platform for Delivering Genome Editing Therapy in CMT
CMTA-funded Yale researchers are testing STEP, a non-viral delivery system for genome-editing medicines in CMT.
Can We Fix the Code? A Look at Genetic Therapies for CMT
Can we fix the genes that cause CMT? CMTA’s Dr. Katherine Forsey breaks down the science behind genetic therapies and what’s heading toward clinical trials.
