LX107

LX107 is an investigational adeno-associated virus (AAV) vector-based gene therapy developed for the treatment of inherited retinal dystrophies (IRD) associated with mutations in the *AIPL1* (Aryl-hydrocarbon interacting protein-like 1) gene. The AIPL1 protein is a specialized chaperone essential for the proper folding and assembly of the phosphodiesterase 6 (PDE6) complex, a key component of the visual phototransduction cascade in photoreceptor cells. Mutations in *AIPL1* typically result in Leber Congenital Amaurosis type 4 (LCA4), a severe form of early-onset blindness, due to the rapid degradation of PDE6 and subsequent photoreceptor cell death. LX107 is designed to be administered via subretinal injection to deliver a functional copy of the *AIPL1* gene to the retina, aiming to restore protein expression, stabilize the PDE6 complex, and preserve or improve visual function in affected patients.

Other names

LX107 injectionLX-107 injectionLX 107 injection