CMTA Addresses the Critical Delivery Challenge Underlying CMT Treatment Development through Four Key Investments

CMTA invests $523,000 in four research projects focused on overcoming treatment delivery barriers in Charcot-Marie-Tooth disease.

GLENOLDEN, PA, UNITED STATES, February 18, 2026 /EINPresswire.com/ -- Charcot-Marie-Tooth Association (CMTA) announced funding for four research projects targeting a fundamental challenge in the development of CMT treatments: delivering therapies to the nerve cells where they can work. Recognizing delivery as essential to turning research into treatments, CMTA is leading the effort to solve this challenge, with these four projects representing the latest phase in this work.

While scientists have made progress understanding the genetic causes of CMT and identifying potential treatments, getting those treatments past the body’s natural defenses and to the affected cells remains unsolved. Peripheral nerves can stretch over three feet from the spine to fingers and toes and are protected by biological barriers designed to keep foreign substances out, including medications. Without solving the delivery issue, promising CMT therapies remain stuck in the lab, unavailable to people who desperately need them.

Through its Strategy To Accelerate Research (CMTA-STAR), CMTA is supporting four complementary approaches to overcome treatment delivery barriers with an investment of $523,000 and related support. Each project targets a different aspect of the challenge, increasing the likelihood of success across all types of CMT:

• Genome-editing delivery: Jianbing Zhou, PhD, at Yale School of Medicine is building a platform to transport genome-editing treatments like CRISPR to cells affected by CMT. This is a crucial step for therapies that aim to correct genetic mutations at their source.

• Blood-nerve barrier crossing: Yizhou Dong, PhD, at the Icahn School of Medicine at Mount Sinai is designing specialized molecules to carry treatments across the blood-nerve barrier, which naturally blocks most therapeutic compounds from reaching peripheral nerves.

• Schwann cell targeting: In collaboration with Shark Tooth Bio and the National Research Council of Canada, Umar Iqbal, PhD, is creating a delivery system that specifically targets Schwann cells, enabling delivery to the cells responsible for myelin production in CMT Type 1.

• Nanoparticle delivery: Alexia Kagiava, PhD, at the Cyprus Institute of Neurology and Genetics, in partnership with the Muscular Dystrophy Association, is engineering therapeutic nanoparticles that protect treatments as they travel through the body and release at the targeted cells.

These four research projects join CMTA’s growing portfolio of delivery science investments. As results from this work emerge, CMTA will assess which approaches show the most promise and where additional investment is needed, ensuring delivery solutions keep pace with scientific progress.

“Delivery continues to be a CMTA research priority,” said Katherine Forsey, PhD, CMTA Chief Research Officer. “Getting treatments to the cells where they are needed has been a significant barrier in CMT, and we’re committed to addressing it. By investing across multiple delivery approaches, we’re helping ensure that when different types of genetic treatments are ready, delivery won’t be an obstacle.”

For more information about CMTA’s delivery science program and other research initiatives to bring relief to people living with CMT, visit cmtausa.org/research.

About Charcot-Marie-Tooth Disease (CMT)

Charcot-Marie-Tooth disease is a group of rare, inheritable peripheral neuropathies. Named after Jean-Martin Charcot, Pierre Marie, and Howard Henry Tooth, who first described it in 1886, CMT causes progressive muscle weakness, loss of sensation, impaired balance, and other debilitating complications. There is currently no cure or approved treatment.

About the Charcot-Marie-Tooth Association (CMTA)

The Charcot-Marie-Tooth Association (CMTA) is the leading nonprofit organization dedicated to improving the lives of people living with Charcot-Marie-Tooth disease (CMT). As the largest philanthropic funder of CMT research, CMTA has invested more than $33 million since 2008 to accelerate research leading to new treatments and ultimately a cure. Through a strategic model that combines targeted funding, specialized scientific tools and resources, and strong patient partnership, CMTA drives promising science toward meaningful outcomes for people living with CMT.

Learn more: cmtausa.org

Kenny Raymond
Charcot-Marie-Tooth Association
+1 800-606-2682
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