SPK-7001

SPK-7001 is an investigational gene therapy developed for the treatment of choroideremia and other inherited retinal dystrophies. It utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver a functional copy of the human CHM gene (encoding Rab escort protein 1, REP1) directly to retinal cells. The therapy aims to restore or supplement CHM protein expression in patients with mutations in the CHM gene, thereby addressing the underlying cause of choroideremia—a progressive X-linked retinal degenerative disease that leads to vision loss. The drug has received orphan drug designation for retinal dystrophies and is being evaluated primarily via subretinal administration in clinical trials[1][2][3][5].

Other names

AAV2-hCHMAAV-2-hCHMAAV 2-hCHM