AAV2-REP1 is a gene therapy used for treating individuals with Choroideremia (CHM). This therapy involves a subretinal injection of a viral vector that carries the gene for Rab Escort Protein-1. The goal is to deliver the correct gene to the retina to help improve or stabilize vision in patients who have previously received this treatment in an earlier study.

AAV8-RPGR is another gene therapy designed for patients with X-Linked Retinitis Pigmentosa (XLRP). This treatment also involves a subretinal injection, but it uses a different viral vector to deliver the RPGR gene. The therapy aims to address the genetic cause of the disease and potentially improve or maintain vision in patients who have been treated with this therapy in a prior study.

Choroideremia – Choroideremia is a rare genetic disorder that primarily affects males and leads to progressive vision loss. It is caused by mutations in the CHM gene, which results in the degeneration of the choroid, retina, and retinal pigment epithelium. The disease typically begins with night blindness in childhood, followed by a gradual loss of peripheral vision. Over time, central vision may also be affected, leading to significant visual impairment. The progression of vision loss varies among individuals, but it generally worsens with age.

X-Linked Retinitis Pigmentosa – X-Linked Retinitis Pigmentosa (XLRP) is a genetic disorder that causes progressive degeneration of the retina, leading to vision loss. It is inherited in an X-linked manner, primarily affecting males, and is associated with mutations in several genes, including RPGR. The condition often begins with night blindness and a gradual loss of peripheral vision during adolescence or early adulthood. As the disease progresses, individuals may experience a narrowing of the visual field and eventual loss of central vision. The rate of progression can vary, but it typically leads to significant visual impairment over time.