timrepigene emparvovec

Timrepigene emparvovec is an investigational gene therapy developed for the treatment of choroideremia, a rare inherited retinal degenerative disease caused by mutations in the CHM gene. The therapy uses a recombinant adeno-associated virus serotype 2 (AAV2) vector to deliver a functional copy of the CHM gene encoding Rab escort protein 1 (REP1) directly to retinal cells via subretinal injection. By restoring REP1 expression, timrepigene emparvovec aims to address the underlying genetic cause of choroideremia, slow or reverse early stages of cell death in retinal cells, and preserve or improve visual function. Clinical trials have shown that some patients experienced stabilization or improvement in visual acuity following treatment[4][5][6][8].

Other names

Recombinant adeno-associated virus expressing REP1