Subretinal Gene Therapy for Wet AMD

RGX-314 (ReGenX Bio) is an experimental subretinal gene therapy designed to neutralize VEGF activity in wet AMD. The one-time therapy uses a proprietary NAV AAV8 platform that offers the potential to alleviate injection burden by increasing the level and duration of gene expression in transduced cells. In this interview from ASRS 2017, Dr. Jeffrey Heier discusses the challenges in designing a surgical trial for a medical disease. Carried out under local anesthesia and standardized across surgeons, the procedure consists of 2 main steps: vitrectomy followed by a subretinal injection. The open-label phase 1 study will test 3 exploratory doses, with primary outcome measures focused on safety and tolerability at 24 weeks. If the study proceeds to phase 2 trials, Dr. Heier anticipates further challenges in designing sham protocols. 

Relevant Financial Disclosures: Dr. Jeffrey Heier receives grant support and is a consultant for ReGenX Bio.