Elpida Therapeutics, Encino, CA
Launch manufacturing of a gene therapy drug for Charcot-Marie-Tooth disease type 4J
CMT Research Foundation funded Elpida Therapeutics to launch manufacturing of a gene therapy drug for Charcot-Marie-Tooth disease type 4J. CMT4J is an ultra-rare subtype, caused by loss of function mutations in the FIG4 gene that result in progressive nerve loss, muscle weakness and loss of sensation. Most people with CMT4J require wheelchair use and multiple surgeries by their teens or early twenties. Respiratory involvement is also common, which can result in ventilatory dependance or early demise. Though rare, a successful gene therapy for CMT4J could help pave the way for the accelerated development of gene therapies for other subtypes of CMT, as well as other neurodegenerative or neuromuscular disorders utilizing a similar technology and approach.
The ELP-02 therapy utilizes an adeno-associated virus vector to intrathecally deliver functional copies of the FIG4 gene into nerve cells lacking normal FIG4 function. A growing body of evidence backs the general safety and effectiveness of AAV vectors.
CMTRF provided $800,000 and CureCMT4J provided $700,000 to Elpida, which will help to cover the costs associated with manufacturing clinical grade ELP-02 doses for the phase I/II trial. Elpida aims to begin treating affected patients in early to mid-2026.
Our ELP-02 gene therapy is trial-ready. Thanks to efforts spearheaded by CureCMT4J, preclinical studies have demonstrated its great potential, and it has earned Investigational New Drug status. Now because of funding from the CMT Research Foundation and CureCMT4J, we’re on the cusp of bringing it to patients.
