Retinitis pigmentosa (XLRP)
Indication: X-linked retinitis pigmentosa (XLRP)
Stage: Proof of concept
Description:
X-linked retinitis pigmentosa (XLRP) is estimated to affect 2.7 to 3.5 males per 100,000, primarily due to mutations in the RPGR gene, the most common cause of XLRP. This severe condition leads to dysfunction of photoreceptor connecting cilia, resulting in progressive degeneration and loss of photoreceptor cells, ultimately causing vision loss. Our therapeutic goal is to deliver normal RPGR gene copies to the retina via an AAV vector to slow or halt this degeneration.