The gene therapy company ViGeneron announced that the first patient has been dosed in its phase 1b clinical trial evaluating intravitreal injection of VG901 to treat retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. The ongoing phase 1b trial is an open-label, single-arm, dose-escalation study investigating the safety, tolerability, and preliminary efficacy of intravitreal VG901, a first-in-class CNGA1 gene therapy for autosomal recessive RP (arRP).
“By delivering a functional CNGA1 gene to retinal photoreceptor target cells, VG901 offers a therapeutic potential in addressing the genetic root cause for patients with retinitis pigmentosa affected by CNGA1 mutations,” Prof. Dr. Katarina Stingl, principal investigator for this trial and head of the Clinic for Hereditary Retinal Degenerations in the Center for Ophthalmology, said in a news release. Mutations in the CNGA1 gene, encoding a subunit of CNG channels in rod photoreceptors, are reported to cause approximately 2% to 8% of arRP.
“Not only is the phase 1b trial designed to provide key insights into the safety and preliminary efficacy of VG901, but also it is a pivotal step in validating our next generation vector platform vgAAV, which has demonstrated superior transduction efficiency and enables intravitreal delivery,” Dr. Caroline Man Xu, ViGeneron’s cofounder and CEO, said in a news release. VG901 has also been granted FDA Orphan Drug Designation status.
