Partnership to advance therapeutic for Charcot-Marie-Tooth disease type 1a

A nonprofit focused on treatments and cures for Charcot-Marie-Tooth disease will partner with a biotechnology firm to advance a novel intrathecal miRNA therapy to address CMT type 1a in adults with the condition.

According to a press release from the CMT Research Foundation, the therapy, developed by Armatus Bio and dubbed ARM-101, will be designed for administration with a single injection to reduce PMP22 overexpression in Schwann cells, a known genetic driver of the disease, and restore daily functionality in patients.

If successful, Armatus is expected to commence clinical trials of ARM-101 in early 2025.

“Armatus Bio is advancing a gene therapy candidate that has shown promising results in early in vitro and animal model studies,” CMT Research Foundation CEO Cleary Simpson said in the release.