News

CMTA Alliance Partner ToolGen announced December 18, 2023 that it received Orphan Drug Designation (ODD) for its rare disease gene correction treatment, TGT-001, from the U.S. Food and Drug Administration (FDA). TGT-001 targets Charcot-Marie-Tooth (CMT), a rare genetic disease related ...

Published: December 11, 2023 French biopharmaceutical company Pharnext SA announced Dec. 10 that the initial results from its Phase III clinical trial (PREMIER trial) for CMT1A did not meet expectations. The company said in a news release that analyzing the results was ...

Novartis is making significant strides in the field of Charcot-Marie-Tooth (CMT) disease treatments, having recently entered into a substantial $1.3 billion deal with Chong Kun Dang Pharmaceutical Corp (CKD). The South Korean pharmaceutical company announced on November 6th that it ...

New technologies in gene editing such as CRISPR/Cas9 present a promising opportunity for the treatment of genetic diseases like CMT at the root cause – often just a single base pair or “letter” change in the DNA sequence. Research lead ...

First Investment from CMTA’s New Venture Philanthropy Arm The Charcot-Marie-Tooth Association (CMTA) today announced that it participated in the Seed Series Extension syndicate for Armatus Bio, Inc. (Armatus). Armatus is an emerging biotechnology company that is advancing a unique gene therapy ...