CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at treating Charcot-Marie-Tooth disease types caused by mutations in the PMP22 gene, which include CMT1A, CMT1E and hereditary neuropathy with liability to...
Since launching their collaboration with CMT Research Foundation in November 2024, researchers at 82VS, the venture studio of Alloy Therapeutics, have made rapid progress and are ahead of schedule testing their novel technology focused on improving RNA therapeutic...
CMT Research Foundation funded partner Augustine Therapeutics recently announced that they completed their Series A financing round and raised $85 million, which will be used to advance their Charcot-Marie-Tooth disease drug candidate, AGT-100216, through a Phase I/II...
The CMT Research Foundation has invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate (AOC) therapeutics for CMT. Oligonucleotide therapeutics offer promise for...
Previous research from Dr. Alessandra Bolino demonstrated that inhibiting a protein called PIKfyve kinase could ameliorate some disease characteristics in CMT4B1 cells. In this project, Dr. Bolino partnered with AcuraStem to test one of their PIKfyve inhibitor drug...
The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a research project to develop new vehicles for delivering gene therapies more efficiently into peripheral nerves. This...
Armatus Bio acquired the CMT1A gene therapy that was developed by Dr. Kleopas Kleopa and Dr. Scott Harper in a previous CMTRF-funded project to advance it further as a therapeutic. To demonstrate this therapy could be a safe and effective treatment for CMT1A, Armatus...
The Charcot-Marie-Tooth Research Foundation participated in the $18.5 million Series A funding round recently announced by Augustine Therapeutics. The financing was led by Asabys Partners, with the participation of Eli Lilly & Company, CMTRF, and current investors...
The CMT Research Foundation has formed a strategic partnership with ReviR Therapeutics to advance small molecule therapeutics that modulate the expression of the gene that causes CMT1A. ReviR Therapeutics is developing a class of drugs called small molecule splice...