GLENOLDEN, PA — The Charcot-Marie-Tooth Association (CMTA) has announced a $240,000 investment in gene replacement therapy targeting CMT4A, a severe form of the disease. This initiative, led by Dr. Xin Chen and Dr. Steven Gray at the University of Texas Southwestern Medical Center, aims to develop a treatment that could extend to other subtypes of Charcot-Marie-Tooth (CMT).
CMT4A is caused by recessive variants in the GDAP1 gene and leads to severe demyelination. The research project will design and test an adeno-associated virus 9 (AAV9) vector. This vector will deliver a functional GDAP1 gene to replace the faulty version. The approach will be tested in models replicating CMT4A conditions. If successful, this method could serve as a template for treating other CMT types, such as CMT4B1 and CMT4D.
“Recent advances in AAV capsids and genome designs have significantly boosted the field of AAV gene therapy,” said Dr. Chen. “With evolving AAV vector technologies, studies on rare forms like CMT4A can provide proof-of-concept for broader CMT therapies.”
Dr. Katherine Forsey, CMTA’s Chief Research Officer, highlighted the strategic importance of this investment. “Through the Strategy to Accelerate Research (STAR) initiative, CMTA has been leading the way in accelerating gene replacement therapies for CMT,” she said. “This investment marks a major step towards developing a roadmap for translating CMT treatments into clinical trials.”
The project leverages advanced AAV vector technologies, aiming to create a blueprint for future therapies. By focusing on these cutting-edge developments, CMTA hopes to bring new treatment options to the broader CMT community.
This investment is part of CMTA’s larger strategy to support impactful research, offering new hope to those affected by various forms of Charcot-Marie-Tooth disease.
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