OliX Pharmaceuticals has treated the first patients in its phase 1 clinical trial evaluating OLX10212, an investigational RNA interference-based gene-silencing (siRNA) therapeutic for the treatment of age-related macular degeneration (AMD), including geographic atrophy (GA) and neovascular (n)AMD. 

The US multicentre, single- and multi-dose, dose-escalation study will evaluate the safety and tolerability of intravitreal injection of OLX10212 in up to 60 patients with nAMD. The asymmetric siRNA technology of OLX10212 targets pathways upstream of VEGF offering increased specificity over conventional siRNA, with the benefit of impermanence over gene therapy, explained Professor Demetrios Vavvas, an Olix scientific advisory board member and Retina Service director at Harvard Medical School. “Through both its mechanism of action and novel target, OLX10212 aims to confer efficacy coupled with extended durability beyond simply blockade of VEGF, addressing key components shared by nAMD and dry AMD.”  

OLX10212 has the potential to not only treat wet AMD, but also decrease the number of treatments needed, added principal investigator Dr Daniel Learned from California Retina Consultants, a participating study site.