Science spotlight: Elixirgen’s full-length dystrophin gene therapy, Insilico’s TNIK inhibitor and more

Elixirgen showed it was able to deliver a linear mRNA encoding a full-length human dystrophin protein, avoiding concerns that miniaturized versions of the gene may lack some critical functionality.

Most of the Duchenne muscular dystrophy (DMD) gene therapies in development deliver only a portion of the dystrophin gene, since it is too large to fit in a conventional adeno-associated viral (AAV) vector.  Elixirgen Therapeutics Inc.’s full-length version was delivered via a nanoparticle, and it restored muscular function in a mouse model of Duchenne muscular dystrophy, as shown at the MDA Clinical & Scientific Conference this week...