ViGeneron GmbH has announced the dosing of the first patient in its Phase 1b clinical trial (NCT06291935) for VG901, a gene therapy targeting retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. This trial represents a significant step forward in addressing the genetic root cause of RP through the delivery of a functional CNGA1 gene to retinal photoreceptor cells.
Participants in the trial will receive a single intravitreal dose of VG901 in their most affected eye, with follow-up monitoring for safety and efficacy over a year. The study includes two cohorts: one receiving a low dose and the other a high dose of VG901, as per the study protocol.
Katarina Stingl, MD, the principal investigator for the trial, expressed enthusiasm about the potential of VG901 to make a meaningful difference in patients' lives. Caroline Man Xu, PhD, co-founder and CEO of ViGeneron, highlighted the importance of this milestone for both the company and patients, noting VG901's FDA Orphan Drug Designation status and the trial's role in validating ViGeneron's next-generation vector platform, vgAAV.
The ongoing Phase 1b clinical trial is an open-label, single-arm, dose-escalation study designed to investigate the safety, tolerability, and preliminary efficacy of VG901. This trial is a crucial step in the development of a potentially transformative therapy for autosomal recessive RP, with the study expected to be completed by the end of 2025.
