Elpida-02

Elpida-02 is an investigational gene therapy developed by Elpida Therapeutics for the treatment of Charcot-Marie-Tooth disease type 4J (CMT4J), a rare progressive neurodegenerative disorder. It is an adeno-associated virus serotype 9 (AAV9) based gene therapy that delivers a codon optimized human FIG4 cDNA coding sequence to target cells. The mechanism of action involves modulation of the FIG4 phosphoinositide 5-phosphatase, aiming to restore or compensate for deficient or dysfunctional FIG4 protein in affected patients. The drug has received orphan drug designation in the US and recently received FDA clearance for its Investigational New Drug (IND) application, enabling initiation of first-in-human Phase 1/2 clinical trials[1][2][3].

Other names

AAV9 gene therapy containing codon optimized human FIG4 cDNA coding sequenceAAV-9 gene therapy containing codon optimized human FIG4 cDNA coding sequenceAAV 9 gene therapy containing codon optimized human FIG4 cDNA coding sequence