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CMTA-STAR Research Projects

Pharmacologic Increase of cGMP for the Treatment of CMT1B/1E Neuropathy

Project announced: August 11, 2026
Updated: August 11, 2026
With CMTA support of $225,720, researchers led by Maurizio D’Antonio, PhD, at Ospedale San Raffaele in Milan, Italy, are evaluating whether a drug repurposing strategy can restore peripheral nerve function in CMT1B and CMT1E. The project will test an FDA-approved phosphodiesterase type 5 (PDE5) inhibitor that raises cyclic GMP (cGMP) levels, along with a therapeuticContinue reading "Pharmacologic Increase of cGMP for the Treatment of CMT1B/1E Neuropathy"
Mr. D'Antonio

Principal Investigator

Maurizio D'Antonio, PhD
Ospedale San Raffaele

Therapy / Approach

Drug Repurposing / Small Molecule

Project Duration

2.5 Years

Total CMTA-STAR Project Investment

$225,720

Active

Hearing and Balance Biomarkers for Outcome Measure Development in CMT1A

Project announced: February 25, 2026
Updated: February 25, 2026
With CMTA support of $141,000, researchers led by Gabriel Corfas, PhD, at the Kresge Hearing Research Institute at Michigan Medicine are conducting a two-year study to examine hearing and balance as potential biomarkers and clinical trial outcome measures in CMT1A. Hearing and balance rely on precise signaling between the inner ear, the peripheral nerves, andContinue reading "Hearing and Balance Biomarkers for Outcome Measure Development in CMT1A"
Professional outdoor headshot of a smiling researcher wearing glasses against a softly blurred green background.

Principal Investigator

Gabriel Corfas, PhD
Michigan Medicine - University of Michigan
Kresge Hearing Research Institute at Michigan Medicine

Therapy / Approach

Biomarkers

Project Duration

2 Years

Total CMTA-STAR Project Investment

$141,000

Active

Schwann Cell-Targeted Peptide-LNPs for Delivery of siRNA Against PMP22 in CMT1A

Project announced: February 18, 2026
Updated: June 12, 2026
With combined support totaling $102,968 from CMTA, Shark Tooth Bio, and the National Research Council Canada (NRC), a team of researchers led by Umar Iqbal, PhD, is conducting a study to develop and test a targeted delivery system designed to help RNA medicines (a type of genetic therapy) reach Schwann cells in CMT1A. Getting medicinesContinue reading "Schwann Cell-Targeted Peptide-LNPs for Delivery of siRNA Against PMP22 in CMT1A"
Umar Iqbal headshot: a man with a gray beard and rectangular glasses, dressed in a gray suit and purple tie, presenting a confident, professional portrait.

Principal Investigator

Umar Iqbal, PhD
NRC Logo
National Research Council Canada/Conseil national de recherches Canada

Therapy / Approach

Delivery

Project Duration

1 Years

Total CMTA-STAR Project Investment

$102,968

Active

Blood–Brain Barrier-Crossing Conjugates for Treating CMT

Project announced: February 18, 2026
Updated: June 30, 2026
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.
Professional headshot of a researcher wearing glasses, a suit jacket, and a collared shirt against a neutral background.

Principal Investigator

Yizhou Dong, PhD
Icahn School of Medicine at Mount Sinai

Therapy / Approach

Delivery

Project Duration

1 Years

Total CMTA-STAR Project Investment

$54,051

Active

Modulating TEAD1 Activity in Charcot-Marie-Tooth Disease Type 1A

Project announced: September 23, 2025
Updated: January 23, 2026
With CMTA support of $281,339, researchers at Albany Medical College, led by Sophie Belin, PhD, and Yannick Poitelon, PhD, are exploring the newly discovered TEAD1 pathway in Schwann cells that may regulate PMP22, the gene at the root of CMT1A.
Sophie Belin, PhD & Yannick Poitelon, PhD

Principal Investigator

Sophie Belin, PhD, & Yannick Poitelon, PhD
Albany Medical College

Therapy / Approach

Small Molecule

Project Duration

3 Years

Total CMTA-STAR Project Investment

$281,339

Active

Augustine Therapeutics – AGT-100216

Project announced: May 30, 2025
Updated: August 19, 2025
The Phase I trial will focus on safety, tolerability, and pharmacokinetics (how the drug moves through the body) in healthy volunteers. Phase I trials are a foundation of clinical research. They help determine whether a new drug is safe enough to move forward into studies involving people with the disease it’s intended to treat.
CMTA Strategy To Accelerate Research (CMTA-STAR) logo on a white background

Principal Investigator

Augustine Therapeutics
Augustine Therapeutics logo featuring a dark navy 'augustine' wordmark with light purple 'therapeutics' text and a starburst graphic, clean modern branding for AGT-100216
Augustine Therapeutics

Therapy / Approach

Clinical Trial - Phase 1

Total CMTA-STAR Project Investment

Unknown

Active

Long Read Sequencing in Undiagnosed Axonal CMT Patients

Project announced: April 22, 2025
Updated: June 12, 2026
The answer is hiding in your DNA. For some, it hasn’t been found yet. This CMTA-funded study uses long-read sequencing to uncover genetic causes of CMT that standard tests may miss.
Mr. Cortese

Principal Investigator

Andrea Cortese, MD, PhD
University College London / National Hospital for Neurology

Therapy / Approach

Gene Identification

Project Duration

2 Years

Total CMTA-STAR Project Investment

$210,000

Active

AAV9-Mediated Gene Therapy to Treat Inherited CMT Neuropathies With Aberrant Myelin

Project announced: April 3, 2025
Updated: March 24, 2026
This CMTA-funded study is testing a gene therapy approach for CMT4B1, a rare form of CMT that affects the myelin around nerves. Early results are encouraging, showing potential to restore nerve health by delivering a working copy of the affected gene.
Ms. Bolino

Principal Investigator

Alessandra Bolino, PhD
IRCCS Ospedale San Raffaele

Therapy / Approach

Genetic Therapy - AAV

Project Duration

3.5 Years

Total CMTA-STAR Project Investment

$442,334

Active

ACT-CMT: CMT1A Natural History Study, Long-Term Progression, and Biomarkers

Project announced: March 31, 2025
Updated: April 3, 2026
With $1.2M in CMTA funding, the ACT-CMT study is tracking long-term CMT1A progression and advancing biomarker development to prepare for future clinical trials.
Mr. Herrmann

Principal Investigator

David Herrmann, MB BCh, MD
University of Rochester and INC sites

Therapy / Approach

Biomarkers / Natural History Study

Project Duration

2 Years

Total CMTA-STAR Project Investment

$1,209,729

Active

Simply CMT