With only one approved gene therapy for RP patients with RPE65 gene mutation, which represents 2% cases of recessive RP and approximately 16% of leber congenital amaurosis (Morimura et al., 1998; Ferrari et al., 2011), the majority are left with supportive care and limited options to significantly improve or prevent RP related vision loss, representing a significant unmet clinical need (O'Neal and Luther, 2024). Here, RPE65 is linked to retinitis pigmentosa 1.