ReviR Therapeutics is developing a class of drugs called small molecule splice modulators, which can regulate the expression of a gene by influencing which components of the genetic code get included in the final version of the RNA transcript. In this project supported by the CMTRF, ReviR Therapeutics will develop a splice modulator that will reduce the expression of the disease gene, which is overexpressed in CMT1A and is the cause of disease symptoms. A benefit of this type of drug is that it can be taken orally, making it much more convenient for patients compared to the injections required for other approaches. The technologies and approaches from ReviR Therapeutics could revolutionize treatment paradigms not only for CMT1A but potentially for other forms of CMT and other neurodegenerative disorders as well.