We are thrilled to see that two CMTRF-funded projects were selected to present their advancements on their CMT gene therapy projects at the ASGCT conference, the premier conference for emerging genetic therapies.


Dr. Lindsay Wallace presented a poster on the CMT1A gene therapy program from the collaborative work between Armatus Bio, Nationwide Children’s Hospital, and the Cypress Institute of Neurology and Genetics. They revealed recent data from their large animal model study which successfully demonstrated that their gene therapy product for CMT1A was able to safely and effectively reach the peripheral nerves of non-human primates and durably reduce the levels of PMP22, the protein over-expressed in CMT1A. These results mark the successful completion of an important milestone for this project in order to move it forward to clinical trials. To see the abstract for this presentation, click here. For more information on this project, click here.

Dr. Daniella Munezero gave a talk on the progress of the CMT1B gene therapy project from Dr. Afrooz Rashnonejad’s laboratory out of Nationwide Children’s Hospital. Dr. Munezero presented encouraging results of their gene therapy approach in both cell and animal models of CMT1B. These exciting results support the continuation of their efforts to optimize their gene therapy product for CMT1B and encourage similar work to be conducted for other subtypes of CMT. To see the abstract for this presentation, click here. For more information on this project, click here.

Both of the projects and their recent advancements presented this week highlight the exciting progress of gene therapies for CMT and encourage more researchers to continue developing and testing these approaches for all types of CMT.

Dr. Lindsay Wallace