EF-210

EF-210 is a preclinical-stage gene therapy developed by EditForce for the treatment of Myotonic Dystrophy Type 1 (DM1). The therapy utilizes a recombinant adeno-associated virus (AAV) vector to deliver a pentatricopeptide repeat (PPR) protein, specifically CUG-PPR1, which is engineered to bind to toxic CUG repeat expansions in the 3' untranslated region (UTR) of the DMPK gene. By binding to these repeats, EF-210 prevents the sequestration of Muscleblind-like (MBNL) proteins, thereby restoring normal RNA splicing and muscle function. This approach addresses the underlying molecular pathology of DM1 by neutralizing the toxic RNA rather than editing the genome directly.

Other names

CUG-PPR1CUG-PPR-1CUG-PPR 1